SAN RAFAEL, Calif., April 10, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Alexander Hardy, President and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, April 24, at 4:30 p.m. ET to discuss first quarter 2024 financial results and provide a general business update. Dial-in Number U.S. / Canada Dial-in Number: 888-330-3556International Dial-in Number: 646-960-0826Conference Call ID: 1816377 U.S. / Canada Replay Dial-in Numbe ...

Although many popular biotech stocks peaked in 2020 and early 2021 and then started to decline, early 2024 has seen signs of recovery and a return of investors’ trust. Nonetheless, this biotech stock powered through the period, achieving miracle growth and maintaining its decades-long tradition of positive returns.The biotech stock in today’s limelight is BioMarin Pharmaceutical (NASDAQ: BMRN), a biotechnology and pharmaceutical company focusing on tackling rare genetic diseases through an innovative portfo ...

Ms. Hubbard Brings More than 20 Years of Experience in the Biopharmaceutical and Diagnostics Industries Appointment Effective May 20, 2024 SAN RAFAEL, Calif., March 7, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that Cristin Hubbard has been named executive vice president and chief commercial officer. Ms. Hubbard will assume responsibility for the company's global commercial operations, effective May 20, succeeding Jeff Ajer. Ms. Hubbard is a proven leader who brings to ...

-       Cowen 44th Annual Healthcare Conference on March 5-       Leerink Partners Global Biopharma Conference on March 11-       Jefferies Biotech on the Bay Summit on March 12-       Barclays 26th Annual Global Healthcare Conference on March 12 SAN RAFAEL, Calif., March 1, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced management is scheduled to participate in four upcoming investor conferences in March. Cowen 44th Annual Healthcare Conference (Boston, MA) Tuesday, March ...

Part I [Business Overview](index=5&type=section&id=Item%201.%20Business) BioMarin is a global biotechnology company focused on developing and commercializing targeted therapies for rare genetic conditions, detailing its product portfolio, R&D, and market dynamics [Overview](index=5&type=section&id=Overview) BioMarin, founded in 1997, is a global biotechnology company developing and commercializing targeted therapies for rare genetic conditions through genetic discovery and robust R&D - BioMarin Pharmaceutical Inc. was founded in **1997**[454](index=454&type=chunk) - The company focuses on developing and commercializing targeted therapies for rare genetic conditions through genetic discovery[454](index=454&type=chunk) - BioMarin has a diverse pipeline of commercial, clinical, and pre-clinical candidates addressing significant unmet medical needs[454](index=454&type=chunk) [Recent Developments](index=5&type=section&id=Recent%20Developments) In 2023, BioMarin achieved **$2.4 billion** in total revenues, driven by VOXZOGO expansion and key approvals for VOXZOGO and ROCTAVIAN in the U.S. and EU - Total revenues for 2023 reached **$2.4 billion**, significantly contributed by VOXZOGO's expansion[455](index=455&type=chunk) - Key approvals in 2023 include U.S. FDA approval for VOXZOGO (children with achondroplasia of all ages with open growth plates) and ROCTAVIAN, and European Commission approval for VOXZOGO (children aged four months and older with open growth plates)[455](index=455&type=chunk) [Commercial Products](index=5&type=section&id=Commercial%20Products) BioMarin markets a portfolio of enzyme and other products for rare genetic disorders, including recently approved ROCTAVIAN, each targeting specific conditions with varying market statuses 2023 Net Product Revenues (in millions of U.S. Dollars) | Commercial Product | Indication | 2023 Net Product Revenues | | :----------------- | :--------- | :------------------------ | | VIMIZIM (elosulfase alpha) | Mucopolysaccharidosis (MPS) IVA | $701.0 | | NAGLAZYME (galsulfase) | MPS VI | $420.3 | | PALYNZIQ (pegvaliase-pqpz) | Phenylketonuria (PKU) | $303.9 | | BRINEURA (cerliponase alfa) | Neuronal ceroid lipofuscinosis type 2 (CLN2) | $161.9 | | ALDURAZYME (laronidase) | MPS I | $131.2 | | VOXZOGO (vosoritide) | Achondroplasia | $469.9 | | KUVAN (sapropterin dihydrochloride) | PKU | $180.8 | - PALYNZIQ is approved in the U.S. for adult PKU patients with uncontrolled blood Phe concentrations (**>600 micromol/L**) and is available only through a REMS program due to anaphylaxis risk[463](index=463&type=chunk)[464](index=464&type=chunk) - VOXZOGO is approved for achondroplasia in children with open growth plates in the U.S. and Japan (all ages) and the EU (four months and older); a pivotal program for hypochondroplasia began in **Q4 2023**, with a treatment study expected mid-2024[6](index=6&type=chunk)[8](index=8&type=chunk) - ROCTAVIAN, a gene therapy for severe hemophilia A, received conditional approval in the EU in **August 2022** and FDA approval in the U.S. in **June 2023**; the European launch is underway, and the company is collaborating on outcome-based reimbursement models[15](index=15&type=chunk)[16](index=16&type=chunk) [Research and Development Programs](index=8&type=section&id=Research%20and%20Development%20Programs) BioMarin has multiple clinical and preclinical product candidates targeting root causes of genetic conditions with significant unmet medical needs, aiming for first-to-market or substantial benefit - The company's development programs focus on addressing root causes of genetic conditions with significant unmet medical needs, aiming for first-to-market or substantial benefit over existing treatments[44](index=44&type=chunk) - In 2023, BioMarin conducted clinical trials and advanced preclinical activities to support Investigational New Drug (IND) or Clinical Trial Application (CTA) submissions[45](index=45&type=chunk) - A strategic portfolio review of all R&D programs commenced in **Q1 2024** to identify assets with the highest potential for patient impact and stockholder value creation[46](index=46&type=chunk) [Manufacturing](index=8&type=section&id=Manufacturing) BioMarin manufactures APIs for several products at its Novato, California, and Shanbally, Cork, Ireland facilities, while outsourcing KUVAN API and managing single-source raw material risks - APIs for ALDURAZYME, NAGLAZYME, PALYNZIQ, VOXZOGO, and ROCTAVIAN are manufactured in Novato, California, which also supports gene therapy development[47](index=47&type=chunk) - APIs for BRINEURA and VIMIZIM are manufactured in Shanbally, Cork, Ireland, which also has aseptic filling and drug product packaging capabilities[47](index=47&type=chunk) - All manufacturing facilities, including third-party contractors, are subject to periodic inspections for cGMP compliance by regulatory authorities[47](index=47&type=chunk)[48](index=48&type=chunk) - The company relies on single-source suppliers for certain raw materials and employs strategies like maintaining higher inventory levels and multiple sourcing to minimize supply risks[49](index=49&type=chunk)[50](index=50&type=chunk) [Sales and Marketing](index=9&type=section&id=Sales%20and%20Marketing) BioMarin maintains commercial organizations and sales forces for direct product support in key markets, utilizing third-party distributors for other regions and Sanofi exclusively for ALDURAZYME - BioMarin has commercial organizations and sales forces for direct product support in the U.S., Europe, South America, and other significant markets[51](index=51&type=chunk) - Sanofi holds the exclusive right to distribute, market, and sell ALDURAZYME globally[52](index=52&type=chunk) - The company utilizes third-party logistics companies for product storage and distribution, and third-party vendors for marketing and sales support services[54](index=54&type=chunk) [Customers](index=9&type=section&id=Customers) BioMarin's customers include specialty pharmacies, hospitals, and government agencies, with three customers accounting for **36%** of 2023 net product revenue, and Sanofi as the sole customer for ALDURAZYME - Customers for BioMarin's products (excluding ALDURAZYME) include specialty pharmacies, hospitals, non-U.S. government agencies, authorized distributors, and pharmaceutical wholesalers[55](index=55&type=chunk) - PALYNZIQ is distributed in the U.S. through a limited number of certified specialty pharmacies via the REMS program[55](index=55&type=chunk) - In 2023, three customers generated **36%** of BioMarin's net product revenue[55](index=55&type=chunk) - The timing of large periodic government orders for NAGLAZYME and VIMIZIM can lead to inconsistent revenue variations quarter-to-quarter[55](index=55&type=chunk) [Competition](index=9&type=section&id=Competition) The biopharmaceutical industry is highly competitive, with BioMarin facing potential new and generic product competition across its disease areas, despite most of its products currently lacking direct approved rivals - The biopharmaceutical industry is rapidly evolving and highly competitive, with numerous companies and research organizations pursuing treatments for the same indications as BioMarin's products[56](index=56&type=chunk) - Except for ROCTAVIAN and KUVAN, BioMarin's products currently have no direct approved competition in the U.S. or EU, but new and generic products are in development[57](index=57&type=chunk) - Generic versions of KUVAN are available in several countries, including multiple versions in the U.S., and manufacturers are challenging BioMarin's KUVAN patent portfolio[12](index=12&type=chunk)[61](index=61&type=chunk) - Potential competition exists across BioMarin's product portfolio, including clinical and preclinical candidates using similar or different treatment strategies for MPS I, MPS VI, MPS IVA, CLN2, PKU, achondroplasia, and severe hemophilia A[59](index=59&type=chunk)[60](index=60&type=chunk)[61](index=61&type=chunk)[62](index=62&type=chunk)[63](index=63&type=chunk) [Patents, Proprietary Rights and Regulatory Exclusivity](index=10&type=section&id=Patents,%20Proprietary%20Rights%20and%20Regulatory%20Exclusivity) BioMarin's success relies on its intellectual property portfolio, including patents and regulatory exclusivity, to protect revenue streams, despite patent expirations for ALDURAZYME and NAGLAZYME - BioMarin maintains and builds its patent portfolio through new filings, prosecution of existing applications, and licensing/acquisition, and protects know-how, trade secrets, and trademarks[65](index=65&type=chunk) - U.S. and most foreign patents are generally effective for **20 years** from the earliest filing date, with potential extensions (e.g., up to **five years** in the U.S. under Hatch-Waxman Act, up to **five years** in the EU via Supplementary Protection Certificates)[67](index=67&type=chunk) - Patents for ALDURAZYME and NAGLAZYME expired in **November 2020** and **November 2023**, respectively[68](index=68&type=chunk) Selected Patent Expiration Dates (Latest) | Product | Territory | Patent Expiration (Latest) | | :------ | :-------- | :------------------------- | | BRINEURA | U.S. | May 5, 2036 | | BRINEURA | EU | May 5, 2036 | | PALYNZIQ | U.S. | February 3, 2031 | | PALYNZIQ | EU | May 23, 2033 | | ROCTAVIAN | US | December 19, 2038 | | ROCTAVIAN | EU | September 10, 2034 | | VIMIZIM | U.S. | January 10, 2032 | | VIMIZIM | EU | July 22, 2031 | | VOXZOGO | U.S. | August 1, 2036 | | VOXZOGO | EU | May 20, 2035 | U.S. Regulatory Exclusivity for Commercial Products | Commercial Products | Biologic Exclusivity Expiration | Orphan Drug Exclusivity Expiration | | :------------------ | :------------------------------ | :--------------------------------- | | BRINEURA | 2029 | 2027 | | PALYNZIQ | 2030 | 2029 | | ROCTAVIAN | 2035 | 2032 | | VIMIZIM | 2026 | 2024 | | VOXZOGO | Not Applicable | 2031 | [Government Regulation](index=12&type=section&id=Government%20Regulation) BioMarin's products are subject to extensive and evolving governmental regulations in the U.S., EU, and other countries, covering all stages from development to commercialization, with non-compliance leading to severe sanctions - BioMarin's products are subject to significant federal, state, local, and non-U.S. regulation across development, manufacture, commercialization, pricing, and reimbursement[79](index=79&type=chunk) - Failure to comply with applicable regulatory requirements can result in severe sanctions, including refusal of approvals, warning letters, product recalls, suspensions, injunctions, fines, and criminal prosecution[81](index=81&type=chunk) [Approval Process in the U.S. and EU](index=13&type=section&id=Approval%20Process%20in%20the%20U.S.%20and%20EU) The drug approval process in the U.S. and EU is lengthy and expensive, involving preclinical tests and multi-phase clinical trials, with the EU's CTR streamlining multi-center trial authorization - The FDA and EU pre-market approval processes typically take many years, involving preclinical laboratory and animal tests (GLP) and adequate human clinical trials (GCP) to establish safety and effectiveness[82](index=82&type=chunk)[83](index=83&type=chunk)[84](index=84&type=chunk) - The EU's Clinical Trials Regulation (CTR), fully applicable from **January 31, 2022**, streamlines the application procedure for clinical trials, allowing a single submission for multi-center trials and shortening regulatory approval deadlines[82](index=82&type=chunk) - Clinical trials typically proceed through three phases: Phase 1 (safety, metabolism), Phase 2 (effectiveness, dosage, adverse effects), and Phase 3 (efficacy and safety in larger patient populations)[85](index=85&type=chunk) - Marketing authorization in the EU can be obtained via a 'Union' MA (centralized procedure by EC/EMA) or 'National MAs' (competent NCAs), with review periods of **210 days** (standard) or **150 days** (accelerated) by the EMA[86](index=86&type=chunk)[88](index=88&type=chunk) [Fast Track Designation and Accelerated Approval](index=14&type=section&id=Fast%20Track%20Designation%20and%20Accelerated%20Approval) The FDA's Fast Track and Accelerated Approval programs expedite development and review for serious conditions, with Accelerated Approval requiring rigorous post-marketing confirmatory trials - The FDA's fast track program facilitates development and expedited review for drugs treating serious or life-threatening conditions with unmet medical needs, allowing for rolling review of NDA/BLA sections[89](index=89&type=chunk)[90](index=90&type=chunk) - Accelerated approval allows drugs for serious illnesses to be approved based on surrogate endpoints, but mandates rigorous post-marketing clinical trials to confirm clinical benefit[91](index=91&type=chunk)[93](index=93&type=chunk) - FDORA authorizes the FDA to require post-approval studies to be underway prior to or within a specified time after approval, with sponsors required to submit progress reports and face enforcement for non-compliance[93](index=93&type=chunk) [Breakthrough Therapy Designation](index=15&type=section&id=Breakthrough%20Therapy%20Designation) The FDA's Breakthrough Therapy program expedites development and review for drugs treating serious or life-threatening diseases with preliminary evidence of substantial improvement over existing therapies - Breakthrough Therapy Designation expedites development and review for drugs treating serious or life-threatening conditions[94](index=94&type=chunk) - This designation is granted when preliminary clinical evidence suggests the drug may offer substantial improvement over existing therapies on one or more clinically significant endpoints[94](index=94&type=chunk) [Adaptive Pathways](index=15&type=section&id=Adaptive%20Pathways) The EMA's Adaptive Pathways provide early and progressive patient access to medicines for high medical needs, including conditional marketing authorization based on less comprehensive data with post-authorization safeguards - The EMA's adaptive pathways allow for early and progressive patient access to medicines for high medical needs, especially in severe diseases where traditional data collection is difficult[95](index=95&type=chunk) - A conditional marketing authorization may be granted with less comprehensive clinical data if the benefit of immediate availability outweighs the inherent risks, subject to post-authorization studies and annual review[96](index=96&type=chunk) [PRIME Program](index=15&type=section&id=PRIME%20Program) The EMA's PRIME program enhances support for developing therapies addressing unmet medical needs, offering early, proactive, and enhanced assistance to optimize data generation and accelerate assessment - The EMA's PRIME program supports the development of therapies targeting unmet medical needs, particularly those offering major therapeutic advantages or for patients with no treatment options[97](index=97&type=chunk) - PRIME offers early, proactive, and enhanced support to drug developers to optimize data generation and facilitate accelerated assessment of drug applications[97](index=97&type=chunk) [Regulation of Approved Products](index=15&type=section&id=Regulation%20of%20Approved%20Products) Approved products are subject to extensive ongoing regulatory requirements by the FDA and European authorities, covering marketing, manufacturing, adverse event reporting, and post-marketing studies, with non-compliance risking approval withdrawal - Marketing approval authorizes commercial marketing with specific prescribing information for approved indications, with strict regulation of post-approval marketing and promotion, including a prohibition on direct-to-consumer advertising for prescription products in the EU[98](index=98&type=chunk)[186](index=186&type=chunk) - Manufacturing facilities and processes are subject to continuous regulation and inspection by the FDA and other agencies to ensure compliance with cGMP regulations[100](index=100&type=chunk)[401](index=401&type=chunk) - Post-marketing requirements include safety and other information submissions, periodic reports, and potentially Risk Evaluation and Mitigation Strategies (REMS) or post-marketing clinical trials to monitor safety and efficacy[399](index=399&type=chunk)[401](index=401&type=chunk) - Product approvals may be withdrawn if regulatory standards are not maintained, problems are identified post-marketing, or if a post-marketing confirmatory trial is unsuccessful[399](index=399&type=chunk)[403](index=403&type=chunk) [Combination Products and Companion Diagnostics](index=16&type=section&id=Combination%20Products%20and%20Companion%20Diagnostics) Combination products and companion diagnostics are subject to specific regulatory requirements, often requiring simultaneous approval and high coordination between drug and device manufacturers - Combination products, defined as products with two or more regulated components (e.g., biologic/drug and device), are subject to specific regulatory requirements, with the FDA designating a primary center for review[101](index=101&type=chunk) - If an in vitro diagnostic is essential for the safe and effective use of a drug or biologic, the FDA generally requires its approval or clearance (known as a companion diagnostic) concurrently with the therapeutic product[102](index=102&type=chunk) - Approval of companion diagnostics often requires a Premarket Approval Application (PMA) and a high level of coordination between the drug/biologic manufacturer and the device manufacturer[102](index=102&type=chunk) [Approval Regulation Outside of the U.S. and the EU](index=17&type=section&id=Approval%20Regulation%20Outside%20of%20the%20U.S.%20and%20the%20EU) Marketing outside the U.S. and EU requires compliance with diverse non-U.S. regulatory requirements for clinical testing and marketing approval, including pricing, coverage, and reimbursement - Marketing outside the U.S. and EU is subject to non-U.S. regulatory requirements for clinical testing and marketing approval, which vary by jurisdiction and may require additional preclinical or clinical testing[19](index=19&type=chunk) - In many countries outside the U.S., approvals for pricing, coverage, and reimbursement by third-party payers (government and private) are also required[19](index=19&type=chunk) [Other Regulation](index=17&type=section&id=Other%20Regulation) BioMarin is subject to various regulations, including biologics and orphan drug exclusivity, healthcare reform initiatives (e.g., IRA), and anti-kickback, false claims, and privacy laws, impacting pricing, reimbursement, and compliance - Biologics in the U.S. receive **12 years** of data exclusivity, preventing biosimilar licensure relying on the reference product data during this period[20](index=20&type=chunk)[404](index=404&type=chunk) - In the EU, new active substances receive eight years of data exclusivity and an additional **two years** of market exclusivity, extendable to **three years** for new therapeutic indications with significant clinical benefit[21](index=21&type=chunk)[405](index=405&type=chunk) - Orphan Drug Designation provides marketing exclusivity for rare diseases (**7 years** in the U.S., **10 years** in the EU, extendable to **12 years** with a Pediatric Investigation Plan), along with other development and regulatory benefits[23](index=23&type=chunk)[406](index=406&type=chunk) - Healthcare reform initiatives, including the EU pharmaceutical legislation review (potential reduction in exclusivity), the PPACA, and the Inflation Reduction Act (IRA) in the U.S. (allowing HHS to negotiate drug prices for Medicare Part B and D, and imposing inflation rebates), significantly impact drug pricing and reimbursement[24](index=24&type=chunk)[210](index=210&type=chunk)[212](index=212&type=chunk)[357](index=357&type=chunk)[361](index=361&type=chunk)[362](index=362&type=chunk)[363](index=363&type=chunk) - The company is subject to federal Anti-Kickback Statute, federal false claims laws, HIPAA, the Physician Payments Sunshine Act, state marketing and promotion laws, and the U.S. Foreign Corrupt Practices Act (FCPA), which prohibit improper payments and require transparency[25](index=25&type=chunk)[27](index=27&type=chunk)[28](index=28&type=chunk)[29](index=29&type=chunk)[30](index=30&type=chunk)[31](index=31&type=chunk) [Pricing and Reimbursement](index=19&type=section&id=Pricing%20and%20Reimbursement) Sales of BioMarin's expensive products depend heavily on coverage and reimbursement from third-party payers, with increasing scrutiny and cost-control measures impacting pricing and commercialization, especially internationally - Sales of BioMarin's expensive products depend significantly on coverage and reimbursement from third-party payers, including government and private insurance plans[32](index=32&type=chunk) - Third-party payers rigorously review and challenge drug prices, medical necessity, and cost-effectiveness, leading to varying reimbursement rates and no assurance of consistent coverage[33](index=33&type=chunk)[34](index=34&type=chunk) - In international markets, reimbursement must be negotiated on a country-by-country basis, often delaying commercial launch, with governments closely regulating drug pricing and employing cost-containment measures like reference pricing and mandatory price cuts[35](index=35&type=chunk) [Government Pricing and Reimbursement Programs for Marketed Drugs in the U.S.](index=20&type=section&id=Government%20Pricing%20and%20Reimbursement%20Programs%20for%20Marketed%20Drugs%20in%20the%20U.S.) BioMarin's U.S. marketed drugs are subject to various government pricing and reimbursement programs, including Medicaid rebates, 340B discounts, and Medicare Part B and D regulations, with the IRA impacting pricing and negotiation - Federal law requires pharmaceutical manufacturers to pay rebates to state Medicaid programs for covered outpatient drugs, with the rebate amount for innovator products being the greater of **23.1%** of AMP or the difference between AMP and best price; the **100%** rebate cap was eliminated effective **January 1, 2024**[357](index=357&type=chunk) - Manufacturers must participate in the 340B drug pricing program, charging certain safety-net healthcare providers no more than an established discounted price for covered outpatient drugs[359](index=359&type=chunk) - Under Medicare Part B, manufacturers report pricing data for separately reimbursable drugs, and the Inflation Reduction Act (IRA) requires quarterly rebates for price increases exceeding inflation[361](index=361&type=chunk) - Medicare Part D requires manufacturers to provide discounts in the coverage gap and, starting in **2025**, subsidize **10-20%** of enrollee prescription costs; the IRA also allows HHS to negotiate selling prices for high-expenditure drugs under Medicare Part B and D, with negotiations for Part D products beginning in **2024**[362](index=362&type=chunk)[363](index=363&type=chunk) [Disclosure of Clinical Trial Information](index=22&type=section&id=Disclosure%20of%20Clinical%20Trial%20Information) Sponsors of FDA-regulated products must disclose clinical trial information, while in the EU, public disclosure of development data, including clinical trial data, is increasing under the CTR - Sponsors of FDA-regulated products must register and disclose clinical trial information, including product, patient population, study sites, and results, which are made public[367](index=367&type=chunk) - In the EU, clinical trial data submitted to the EMA, including preclinical and patient-level data, is increasingly subject to public disclosure under the Clinical Trials Information System (CTIS) established by the CTR, with confidentiality exceptions interpreted narrowly[367](index=367&type=chunk) [Pediatric Indications](index=22&type=section&id=Pediatric%20Indications) In the U.S., PREA requires pediatric data for NDAs/BLAs, with BPCA offering exclusivity extensions, while in the EU, PIP compliance can lead to extensions of supplementary protection certificates or orphan market exclusivity - Under the Pediatric Research Equity Act (PREA) in the U.S., most NDAs or BLAs must include data to assess safety and effectiveness in relevant pediatric subpopulations, with deferrals or waivers possible[368](index=368&type=chunk) - The Best Pharmaceuticals for Children Act (BPCA) provides an additional **six-month** period of market exclusivity for sponsors who submit results of FDA-requested pediatric studies within required timeframes[368](index=368&type=chunk) - In the EU, companies must agree to a Pediatric Investigation Plan (PIP) and conduct pediatric clinical trials; compliance makes products eligible for a **six-month** extension of supplementary protection certificates or a **two-year** extension of orphan market exclusivity[37](index=37&type=chunk) [Privacy and Security Legislation](index=22&type=section&id=Privacy%20and%20Security%20Legislation) BioMarin is subject to a continually evolving global landscape of data privacy and security obligations, including U.S. federal and state laws, Canadian PIPEDA, and European GDPR, with potential for significant fines for non-compliance - BioMarin is subject to numerous data privacy and security obligations, including federal, state, local, and foreign laws such as the Federal Trade Commission Act, California Consumer Privacy Act (CCPA), California Privacy Rights Act (CPRA), Canadian Personal Information Protection and Electronic Documents Act, EU's General Data Protection Regulation (GDPR), and UK GDPR[38](index=38&type=chunk)[39](index=39&type=chunk)[41](index=41&type=chunk)[43](index=43&type=chunk) - The CCPA and CPRA impose obligations on businesses regarding disclosures, consumer rights (access, delete, opt-out), and provide for civil penalties and a private right of action for data breaches[39](index=39&type=chunk)[41](index=41&type=chunk) - The GDPR imposes strict obligations on personal data processing, including requirements for specified purposes, adequacy, security, transfer restrictions, retention limits, breach notification, and data subject rights, with potential fines up to **€20 million** or **4%** of annual global revenue[43](index=43&type=chunk)[342](index=342&type=chunk) - The legislative and regulatory environments are continually evolving, with new laws and regulations being enacted or proposed in various jurisdictions, increasing compliance burdens and potential liabilities[39](index=39&type=chunk)[42](index=42&type=chunk)[343](index=343&type=chunk) [Human Capital](index=23&type=section&id=Human%20Capital) As of December 31, 2023, BioMarin had **3,401** employees worldwide, committed to Diversity, Equity, and Inclusion (DEI), offering competitive compensation, comprehensive benefits, and professional growth opportunities - As of **December 31, 2023**, BioMarin had **3,401** employees worldwide, with **1,509** in operations, **807** in research and development, **535** in sales and marketing, and **550** in administration[114](index=114&type=chunk) - The company is committed to Diversity, Equity, and Inclusion (DEI) across all aspects of its organization, with **49%** racial and ethnic minorities in the U.S. and **51%** women globally, including **52%** of director-level and above positions held by women as of **December 31, 2023**[122](index=122&type=chunk) - BioMarin offers competitive compensation and benefits, including market-competitive salary, bonuses, equity, healthcare, retirement plans, paid time off, and wellness programs, and conducts regular pay equity assessments[125](index=125&type=chunk)[126](index=126&type=chunk) - The company invests in employee career growth through various development opportunities, including training, mentoring, coaching, and tuition reimbursement, and supports patient and local communities through outreach programs and scholarships[127](index=127&type=chunk)[128](index=128&type=chunk) [Other Information](index=24&type=section&id=Other%20Information) BioMarin Pharmaceutical Inc., incorporated in Delaware in **October 1996**, with principal offices in San Rafael, California, makes its SEC filings available free of charge on its website and the SEC's website - BioMarin Pharmaceutical Inc. was incorporated in Delaware in **October 1996**[129](index=129&type=chunk) - The company's principal executive offices are located at **770 Lindaro Street, San Rafael, California 94901**[129](index=129&type=chunk) - Annual reports on Form 10-K, quarterly reports on Form 10-Q, proxy statements, and other SEC filings are available free of charge on www.bmrn.com and www.sec.gov[129](index=129&type=chunk) [Risk Factors](index=25&type=section&id=Item%201A.%20Risk%20Factors) This section outlines significant risks, categorized into business, regulatory, financial, manufacturing, international, intellectual property, and ownership of securities, that could adversely affect BioMarin's business, financial condition, or stock price - An investment in BioMarin's securities involves a high degree of risk due to operating in a dynamic and rapidly changing industry[131](index=131&type=chunk) - Key risk categories include Business and Operational, Regulatory, Financial and Financing, Manufacturing, International Operations, Intellectual Property, and Risks Related to Ownership of Securities[132](index=132&type=chunk)[169](index=169&type=chunk)[225](index=225&type=chunk)[241](index=241&type=chunk)[269](index=269&type=chunk)[294](index=294&type=chunk)[306](index=306&type=chunk) [Business and Operational Risks](index=25&type=section&id=Business%20and%20Operational%20Risks) BioMarin faces significant business and operational risks, including challenges in securing adequate coverage and reimbursement for expensive products, intense competition, and potential delays in development or issues with collaborations - Failure to obtain and maintain adequate coverage and reimbursement for expensive products, especially gene therapies, poses a significant risk to sales and commercial viability, as high per-patient prices are necessary for profitability due to small target populations[133](index=133&type=chunk)[134](index=134&type=chunk)[140](index=140&type=chunk)[145](index=145&type=chunk) - Gene therapy products like ROCTAVIAN present unique commercial risks, including uncertainty in pricing, coverage, and reimbursement, potential repayments under outcomes-based agreements, and challenges in gaining public and medical community acceptance due to novelty and potential for non-durable effects or irreversible side effects[141](index=141&type=chunk)[142](index=142&type=chunk)[143](index=143&type=chunk)[144](index=144&type=chunk) - The company faces intense competition from more effective or less expensive products, faster regulatory approvals by competitors, and the adverse impact of generic versions of KUVAN on revenues[147](index=147&type=chunk)[148](index=148&type=chunk)[155](index=155&type=chunk)[156](index=156&type=chunk) - Risks include delays or failure to achieve projected development goals, which can impact commercialization and management credibility, and potential negative outcomes from licensing arrangements or activist investor actions that can divert resources and disrupt strategic plans[157](index=157&type=chunk)[158](index=158&type=chunk)[161](index=161&type=chunk)[162](index=162&type=chunk) [Regulatory Risks](index=28&type=section&id=Regulatory%20Risks) BioMarin faces substantial regulatory risks, including potential delays or failure to obtain marketing approval, extensive ongoing post-approval requirements, costly and uncertain clinical trials, and the impact of government price controls and healthcare reforms - Obtaining regulatory approval to market and sell product candidates is a lengthy, expensive, and uncertain process, with non-U.S. approvals varying and potentially requiring additional clinical testing[163](index=163&type=chunk)[164](index=164&type=chunk)[170](index=170&type=chunk)[172](index=172&type=chunk) - Approved products are subject to extensive ongoing regulatory requirements, including manufacturing standards (cGMP), post-approval clinical research, product labeling, advertising, and promotional activities, with non-compliance leading to penalties, restrictions, or withdrawal of marketing approval[179](index=179&type=chunk)[180](index=180&type=chunk)[181](index=181&type=chunk)[184](index=184&type=chunk)[185](index=185&type=chunk)[186](index=186&type=chunk)[187](index=187&type=chunk)[188](index=188&type=chunk)[191](index=191&type=chunk) - Preclinical studies and clinical trials are costly, lengthy, and highly uncertain, with preliminary or interim data not necessarily predictive of final results, and adverse or inconclusive outcomes potentially preventing regulatory approval[192](index=192&type=chunk)[193](index=193&type=chunk)[194](index=194&type=chunk)[195](index=195&type=chunk) - Government price controls, healthcare reforms (such as the Inflation Reduction Act in the U.S. and cost-containing measures in the EU), and the potential for biosimilar competition or loss of orphan drug exclusivity could restrict pricing, reduce revenues, and adversely affect operations[198](index=198&type=chunk)[199](index=199&type=chunk)[200](index=200&type=chunk)[201](index=201&type=chunk)[204](index=204&type=chunk)[206](index=206&type=chunk)[209](index=209&type=chunk)[210](index=210&type=chunk)[212](index=212&type=chunk)[213](index=213&type=chunk)[214](index=214&type=chunk)[217](index=217&type=chunk)[218](index=218&type=chunk)[219](index=219&type=chunk)[221](index=221&type=chunk) [Financial and Financing Risks](index=36&type=section&id=Financial%20and%20Financing%20Risks) BioMarin faces financial and financing risks, including potential continued operating losses, the need for additional capital, and substantial indebtedness of **$1.1 billion** as of **December 31, 2023**, which may limit flexibility and increase vulnerability - BioMarin has operated at a net loss for most years since inception and may incur future operating losses or be unable to sustain positive cash flows, potentially forcing a reduction in operations[226](index=226&type=chunk)[227](index=227&type=chunk) - Additional financing may be required to fund debt repayment, future milestone payments, ongoing operations, preclinical studies, clinical trials, and potential licenses and acquisitions[228](index=228&type=chunk)[230](index=230&type=chunk) - As of **December 31, 2023**, BioMarin had **$1.1 billion** (undiscounted) in principal amount of indebtedness, including **$495.0 million** in 2024 Notes and **$600.0 million** in 2027 Notes, which will require cash repayment if not converted[229](index=229&type=chunk)[236](index=236&type=chunk) - This indebtedness may limit the company's ability to borrow additional funds, require a substantial portion of cash flow for debt service, reduce flexibility, and increase vulnerability to adverse economic conditions[236](index=236&type=chunk) - The ability to refinance outstanding indebtedness depends on capital markets and the company's financial condition at the time, with no assurance of commercially reasonable terms or availability[239](index=239&type=chunk)[240](index=240&type=chunk) [Manufacturing Risks](index=38&type=section&id=Manufacturing%20Risks) BioMarin's manufacturing operations are subject to strict cGMP regulations, with non-compliance risking delays and fines, and reliance on single-source suppliers and limited facilities creating significant supply interruption risks - Failure to comply with manufacturing regulations (cGMP) can lead to delays in product candidate approval, warning letters, fines, product recalls, production suspensions, and enforcement actions[242](index=242&type=chunk)[243](index=243&type=chunk)[245](index=245&type=chunk) - Developing and maintaining manufacturing processes for sufficient quantities at acceptable costs is difficult, especially for complex gene therapy products, which are based on relatively novel technology and present significant technical, scientific, and capital challenges[246](index=246&type=chunk)[247](index=247&type=chunk)[249](index=249&type=chunk)[251](index=251&type=chunk) - Reliance on single-source suppliers for critical raw materials and a limited number of manufacturing facilities exposes the company to supply interruptions due to various factors, including labor issues, changes in sources, and raw material costs[256](index=256&type=chunk)[257](index=257&type=chunk)[264](index=264&type=chunk)[265](index=265&type=chunk) - Termination of the Manufacturing, Marketing and Sales Agreement with Sanofi for ALDURAZYME could prevent or significantly delay/diminish BioMarin's ability to commercialize the product[260](index=260&type=chunk)[261](index=261&type=chunk)[266](index=266&type=chunk)[267](index=267&type=chunk)[268](index=268&type=chunk) [Risks Related to International Operations](index=40&type=section&id=Risks%20Related%20to%20International%20Operations) BioMarin's significant international operations expose it to increased complexity, diverse regulatory requirements, geopolitical instability, diminished intellectual property protection, currency risks, and compliance challenges with anti-corruption laws - International operations subject BioMarin to increased complexity, diverse regulatory requirements, geopolitical instability (e.g., Russia's invasion of Ukraine impacting sales and receivables), diminished intellectual property protection, and trade protection measures[270](index=270&type=chunk)[271](index=271&type=chunk)[273](index=273&type=chunk) - A significant portion of international sales are made through special access programs, and changes to these programs (requirements, funding, classification) could adversely affect product sales and revenues[275](index=275&type=chunk)[276](index=276&type=chunk)[277](index=277&type=chunk)[278](index=278&type=chunk) - International operations pose currency risks, as a significant portion of revenues and expenses are in foreign currencies, impacting operating results and net income, despite hedging strategies[279](index=279&type=chunk)[280](index=280&type=chunk)[281](index=281&type=chunk)[282](index=282&type=chunk) - Compliance with U.S. export control and economic sanctions (e.g., OFAC sanctions on Russia, Belarus, Iran) and anti-corruption legislation (e.g., FCPA) is required, with failure potentially resulting in fines, criminal penalties, and adverse effects on business and reputation[283](index=283&type=chunk)[284](index=284&type=chunk)[285](index=285&type=chunk)[286](index=286&type=chunk)[287](index=287&type=chunk)[288](index=288&type=chunk)[289](index=289&type=chunk)[290](index=290&type=chunk) [Intellectual Property Risks](index=43&type=section&id=Intellectual%20Property%20Risks) BioMarin's success depends on protecting its intellectual property, but patent protection is complex and uncertain, trade secrets are vulnerable, and public disclosure of clinical trial data in the EU could compromise proprietary information - The patent positions of biopharmaceutical products are complex and uncertain, particularly for products where key information has been in the public domain, potentially hindering the ability to obtain or enforce strong patent protection[296](index=296&type=chunk)[297](index=297&type=chunk) - Patents have limited duration, are expensive and time-consuming to enforce, and can be challenged by competitors on grounds of prior invention, lack of originality, novelty, or obviousness[298](index=298&type=chunk)[303](index=303&type=chunk) - Trade secrets are vulnerable to unintentional or willful disclosure by employees, consultants, or contractors, and enforcement is expensive, time-consuming, and has unpredictable outcomes, especially outside the U.S.[299](index=299&type=chunk) - Public disclosure of clinical trial information and MAA data in the EU can adversely affect BioMarin's business by revealing confidential methodologies, preventing IP protection for innovations, and enabling competitors[300](index=300&type=chunk)[302](index=302&type=chunk) - Competitors and other third parties may possess intellectual property that could limit BioMarin's ability to market and commercialize its products, potentially leading to infringement lawsuits, substantial damages, injunctions, or the need for costly licenses or product redesigns[304](index=304&type=chunk)[310](index=310&type=chunk)[311](index=311&type=chunk) [Risks Related to Ownership of Our Securities](index=44&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Securities) BioMarin's stock price may remain volatile, influenced by various factors, and the conversion of convertible notes could dilute existing stockholders, while anti-takeover provisions may hinder acquisitions - BioMarin's stock price has been and may in the future be volatile, influenced by factors such as product sales, R&D progress, regulatory actions, competition, economic conditions, and analyst assessments[312](index=312&type=chunk)[313](index=313&type=chunk) - The company has been subject to securities class action litigation in the past (e.g., related to ROCTAVIAN and BMN 307), which can result in substantial costs and divert management's attention[307](index=307&type=chunk)[391](index=391&type=chunk)[394](index=394&type=chunk) - The conversion of outstanding convertible notes will dilute the ownership interests of existing stockholders and may depress the price of common stock; the fundamental change repurchase feature of the notes may also delay or prevent beneficial takeover attempts[308](index=308&type=chunk)[314](index=314&type=chunk)[315](index=315&type=chunk)[317](index=317&type=chunk) - Anti-takeover provisions in charter documents and under Delaware law, along with exclusive forum provisions in the bylaws, may make an acquisition of the company more difficult and could limit stockholders' ability to obtain a favorable judicial forum for certain disputes[317](index=317&type=chunk)[318](index=318&type=chunk)[319](index=319&type=chunk)[320](index=320&type=chunk)[321](index=321&type=chunk)[323](index=323&type=chunk) [General Risk Factors](index=47&type=section&id=General%20Risk%20Factors) BioMarin's general risks include dependence on key personnel, challenges of managing growth, adverse impacts from tax law changes, penalties from non-compliance with healthcare laws, product liability, cybersecurity vulnerabilities, and macroeconomic factors - BioMarin's future growth and success depend heavily on its ability to attract, retain, manage, and motivate qualified employees, including senior management, and to effectively manage its growth, especially for products targeting larger patient populations[106](index=106&type=chunk)[325](index=325&type=chunk)[326](index=326&type=chunk)[327](index=327&type=chunk) - New tax laws or regulations, or adverse interpretations of existing ones (e.g., U.S. R&D capitalization requirements, OECD BEPS 2.0 Pillar Two proposals), could increase costs and materially adversely affect the company's business and financial condition[328](index=328&type=chunk)[329](index=329&type=chunk)[330](index=330&type=chunk)[331](index=331&type=chunk) - Violations of healthcare laws (e.g., federal Anti-Kickback Statute, false claims laws, HIPAA, Physician Payments Sunshine Act, state laws) or privacy and data protection laws (e.g., CCPA/CPRA, GDPR) could result in significant penalties, regulatory scrutiny, and exclusion from government healthcare programs[108](index=108&type=chunk)[332](index=332&type=chunk)[333](index=333&type=chunk)[334](index=334&type=chunk)[335](index=335&type=chunk)[336](index=336&type=chunk)[338](index=338&type=chunk)[339](index=339&type=chunk)[340](index=340&type=chunk)[341](index=341&type=chunk)[342](index=342&type=chunk)[345](index=345&type=chunk)[346](index=346&type=chunk) - The company is exposed to product liability risks, with potential for substantial liabilities that may exceed insurance coverage, and relies significantly on information technology systems, making it vulnerable to cybersecurity incidents, disruptions, and risks associated with implementing a new global ERP system[111](index=111&type=chunk)[118](index=118&type=chunk)[347](index=347&type=chunk)[348](index=348&type=chunk)[349](index=349&type=chunk)[350](index=350&type=chunk)[351](index=351&type=chunk)[352](index=352&type=chunk)[353](index=353&type=chunk) - Various macroeconomic factors, including inflation, interest rates, foreign currency exchange rates, natural disasters (e.g., earthquakes in California facilities), geopolitical instability, and global public health threats, could adversely affect business operations, financial condition, and results[113](index=113&type=chunk)[119](index=119&type=chunk)[120](index=120&type=chunk)[355](index=355&type=chunk)[356](index=356&type=chunk)[372](index=372&type=chunk)[374](index=374&type=chunk)[375](index=375&type=chunk)[376](index=376&type=chunk)[377](index=377&type=chunk) [Unresolved Staff Comments](index=53&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) There are no unresolved staff comments to report - There are no unresolved staff comments[379](index=379&type=chunk) [Cybersecurity](index=53&type=section&id=Item%201C.%20Cybersecurity) BioMarin's cybersecurity risk management program leverages the NIST framework, with Board oversight and internal committees, and has not experienced a material cybersecurity incident to date - BioMarin's cybersecurity risk management program leverages the National Institute of Standards and Technology (NIST) cybersecurity framework[380](index=380&type=chunk) - The company implements various technical, physical, and organizational measures, including policies, standards, processes, and technical capabilities, to manage and mitigate material risks from cybersecurity threats[381](index=381&type=chunk) - Cybersecurity risk management is integrated into the company's overall risk management processes, with regular testing and assistance from third-party security service providers and consultants[382](index=382&type=chunk)[383](index=383&type=chunk) - Cybersecurity governance includes oversight by the Board of Directors and Audit Committee, an Executive Cybersecurity Committee (ECC) for program oversight and incident materiality, and a Cybersecurity Risk Committee (CRC) for enterprise-wide risk management[385](index=385&type=chunk)[387](index=387&type=chunk)[388](index=388&type=chunk) - As of the date of the Annual Report on Form 10-K, BioMarin has not experienced a cybersecurity incident that resulted in a material adverse impact to its business or operations[384](index=384&type=chunk) [Properties](index=54&type=section&id=Item%202.%20Properties) BioMarin owns and leases facilities in California and Ireland for corporate, manufacturing, laboratory, office, and warehouse use, which are expected to be adequate for foreseeable operations Significant Owned and Leased Properties (as of December 31, 2023) | Location | Approximate Square Feet | Use | Lease Expiration Date | | :------- | :---------------------- | :-- | :-------------------- | | San Rafael facility, San Rafael, California | 407,300 | Corporate headquarters, laboratory and office | Owned property | | Several facilities in Novato, California | 293,300 | Clinical and commercial manufacturing, laboratory and office | Owned property | | Several leased facilities in Novato, California | 158,600 | Office and warehouse | 2027 | | Shanbally facility, Cork, Ireland | 260,700 | Manufacturing, laboratory and office | Owned property | - The company expects its current properties, including owned and leased facilities, to be adequate for its operations for the foreseeable future[390](index=390&type=chunk) [Legal Proceedings](index=54&type=section&id=Item%203.%20Legal%20Proceedings) BioMarin has been involved in shareholder class action lawsuits (one settled, one dismissed) and derivative actions (settlement pending), and is cooperating with a U.S. Department of Justice subpoena regarding sponsored testing programs - A shareholder class action lawsuit filed in **September 2020**, alleging misleading statements regarding ROCTAVIAN's clinical trials and BLA, was settled and received final court approval on **November 14, 2023**[391](index=391&type=chunk)[393](index=393&type=chunk)[896](index=896&type=chunk) - A second shareholder class action lawsuit filed in **October 2021**, alleging misleading statements about BMN 307's safety profile and commercial prospects, was dismissed with prejudice by the district court in **January 2023**, and this dismissal was affirmed by the Ninth Circuit on **February 15, 2024**[394](index=394&type=chunk) - Two shareholder derivative actions filed in **January** and **May 2023**, asserting breach of fiduciary duty claims related to the ROCTAVIAN class action, have a stipulation of settlement pending final court approval[395](index=395&type=chunk) - BioMarin received a subpoena from the U.S. Department of Justice requesting documents regarding sponsored testing programs for VIMIZIM and NAGLAZYME, and is cooperating fully with the investigation[333](index=333&type=chunk)[876](index=876&type=chunk) [Mine Safety Disclosures](index=55&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to BioMarin Pharmaceutical Inc - The item on Mine Safety Disclosures is not applicable[396](index=396&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=56&type=section&id=Item%205.%20Market%20for%20Registrant's%20Common%20Equity,%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) BioMarin's common stock is listed on the Nasdaq Global Select Market under "BMRN," with no cash dividends anticipated, and **188,675,622** shares outstanding as of **February 16, 2024** - BioMarin's common stock is listed on the Nasdaq Global Select Market under the symbol "**BMRN**"[411](index=411&type=chunk) - The company has never paid cash dividends on its common stock and does not anticipate paying them in the foreseeable future[411](index=411&type=chunk) - As of **February 16, 2024**, there were **34** holders of record for **188,675,622** outstanding shares of common stock[414](index=414&type=chunk) - There were no recent sales of unregistered securities or issuer purchases of equity securities[412](index=412&type=chunk)[413](index=413&type=chunk) [Performance Graph](index=57&type=section&id=Performance%20Graph) The performance graph illustrates the historical value of a **$100** investment in BioMarin common stock compared to selected indices from **2018** to **2023**, cautioning that past performance is not indicative of future results Comparative Stock Performance (Value of $100 invested on December 31, 2018) | Index/Stock | 2018 | 2019 | 2020 | 2021 | 2022 | 2023 | | :---------- | :--- | :--- | :--- | :--- | :--- | :--- | | BioMarin Pharmaceutical Inc. | $100.00 | $99.30 | $102.98 | $103.76 | $121.54 | $113 | | Nasdaq Composite Index | $100.00 | $136.69 | $198.10 | $242.03 | $163.28 | $236 | | Nasdaq Biotechnology | $100.00 | $125.11 | $158.17 | $158.20 | $142.19 | $148 | | SPDR S&P Biotech ETF | $100.00 | $132.56 | $196.63 | $156.42 | $115.96 | $124 | - The comparisons shown in the graph are based upon historical data and are not indicative of, nor intended to forecast, the potential future performance of BioMarin's stock[417](index=417&type=chunk) [Reserved](index=57&type=section&id=Item%206.%20%5BReserved%5D) This item is reserved and contains no information - Item 6 is reserved[419](index=419&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=58&type=section&id=Item%207.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's perspective on BioMarin's financial condition and results, highlighting **$2.4 billion** in total revenues and **$167.6 million** net income in **2023**, driven by VOXZOGO, while managing macroeconomic risks and advancing the pipeline - The MD&A helps readers understand BioMarin's results of operations and financial condition, supplementing the audited Consolidated Financial Statements[420](index=420&type=chunk) - Conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, and supply chain disruptions, could impact global revenue sources and overall business operations[424](index=424&type=chunk) - In **2023**, BioMarin achieved **$2.4 billion** in total revenues, with significant contributions from VOXZOGO expansion, and made important advancements in its product development pipeline, including key FDA and EC approvals[426](index=426&type=chunk) [Overview](index=58&type=section&id=Overview_58) BioMarin, founded in **1997**, is a global biotechnology company focused on genetic discovery, developing and commercializing targeted therapies for rare genetic conditions, with a diverse pipeline - BioMarin Pharmaceutical Inc. was founded in **1997** as a global biotechnology company[421](index=421&type=chunk) - The company develops and commercializes targeted therapies for rare genetic conditions, leveraging robust research and development capabilities[421](index=421&type=chunk) Commercial Products as of December 31, 2023 | Product | Indication | | :------ | :--------- | | VIMIZIM (elosulfase alpha) | Mucopolysaccharidosis (MPS) IVA | | NAGLAZYME (galsulfase) | MPS VI | | PALYNZIQ (pegvaliase-pqpz) | Phenylketonuria (PKU) | | BRINEURA (cerliponase alfa) | Neuronal ceroid lipofuscinosis type 2 (CLN2) | | ALDURAZYME (laronidase) | MPS I | | VOXZOGO (vosoritide) | Achondroplasia | | KUVAN (sapropterin dihydrochloride) | PKU | | ROCTAVIAN (valoctocogene roxaparvovec) | Severe Hemophilia A | [2023 Financial Highlights](index=59&type=section&id=2023%20Financial%20Highlights) BioMarin reported total revenues of **$2,419.2 million** in **2023**, an increase from **$2,096.0 million** in **2022**, with net income rising to **$167.6 million** in **2023** from **$141.6 million** in **2022** Key Financial Data (Twelve Months Ended December 31, in millions of U.S. Dollars) | Metric | 2023 | 2022 | 2021 | | :----- | :--- | :--- | :--- | | Total revenues | $2,419.2 | $2,096.0 | $1,846.3 | | Cost of sales | $514.9 | $483.7 | $470.5 | | Research and development (R&D) expense | $746.8 | $649.6 | $628.8 | | Selling, general and administrative (SG&A) expense | $937.3 | $854.0 | $759.4 | | Gain on sale of nonfinancial assets, net | $— | $108.0 | $— | | Provision for (benefit from) income taxes | $20.9 | $8.0 | $(11.3) | | Net income (loss) | $167.6 | $141.6 | $(64.1) | [Results of Operations](index=60&type=section&id=Results%20of%20Operations) This section details changes in BioMarin's financial performance, including increased Net Product Revenues driven by VOXZOGO and VIMIZIM, improved gross margin, rising R&D and SG&A expenses, and changes in other income/expense items [Net Product Revenues](index=60&type=section&id=Net%20Product%20Revenues) Net Product Revenues increased by **$330.5 million** in **2023**, primarily due to higher sales volume of VOXZOGO and VIMIZIM, partially offset by declines in KUVAN and NAGLAZYME, with foreign currency having an unfavorable **$100.0 million** impact Net Product Revenues (in millions of U.S. Dollars) | Product | 2023 | 2022 | 2021 | 2023 vs. 2022 | 2022 vs. 2021 | | :------ | :--- | :--- | :--- | :------------ | :------------ | | VIMIZIM | $701.0 | $663.8 | $623.1 | $37.2 | $40.7 | | NAGLAZYME | $420.3 | $443.8 | $380.4 | $(23.5) | $63.4 | | PALYNZIQ | $303.9 | $255.0 | $237.5 | $48.9 | $17.5 | | BRINEURA | $161.9 | $154.3 | $128.0 | $7.6 | $26.3 | | ALDURAZYME | $131.2 | $128.4 | $122.8 | $2.8 | $5.6 | | **Total enzyme product revenues** | **$1,718.3** | **$1,645.3** | **$1,491.8** | **$73.0** | **$153.5** | | VOXZOGO | $469.9 | $169.1 | $5.9 | $300.8 | $163.2 | | KUVAN | $180.8 | $227.6 | $285.8 | $(46.8) | $(58.2) | | ROCTAVIAN | $3.5 | $— | $— | $3.5 | $— | | **Total net product revenues** | **$2,372.5** | **$2,042.0** | **$1,783.5** | **$330.5** | **$258.5** | - The increase in Net Product Revenues in **2023** was primarily driven by higher sales volume of VOXZOGO due to new patients and VIMIZIM due to new patients and timing of large government orders[428](index=428&type=chunk) - The increase was partially offset by lower KUVAN sales due to generic competition and lower NAGLAZYME sales due to timing of large government orders[428](index=428&type=chunk) - The unfavorable impact of foreign currency exchange rates on USD reported results in **2023** was **$100.0 million**, primarily driven by the Argentine Peso, Euro, Japanese Yen, and Russian Ruble[470](index=470&type=chunk) [Royalty and Other Revenues](index=61&type=section&id=Royalty%20and%20Other%20Revenues) Royalty and Other Revenues decreased by **$7.3 million** in **2023** compared to **2022**, primarily due to lower royalty revenues earned from third parties, with future royalties still expected Royalty and Other Revenues (in millions of U.S. Dollars) | Metric | 2023 | 2022 | 2021 | 2023 vs. 2022 | 2022 vs. 2021 | | :----- | :--- | :--- | :--- | :------------ | :------------ | | Royalty and other revenues | $46.7 | $54.0 | $62.8 | $(7.3) | $(8.8) | - The decrease in Royalty and Other Revenues in **2023** compared to **2022** was primarily due to lower royalty revenues earned from third parties[472](index=472&type=chunk) [Cost of Sales and Gross Margin](index=61&type=section&id=Cost%20of%20Sales%20and%20Gross%20Margin) Cost of Sales increased in **2023** due to higher sales volumes, while gross margin improved to **78.7%** from **76.9%** due to a favorable product mix towards higher-margin VOXZOGO and lower per-unit manufacturing costs Cost of Sales and Gross Margin (in millions of U.S. Dollars) | Metric | 2023 | 2022 | 2021 | 2023 vs. 2022 | 2022 vs. 2021 | | :----- | :--- | :--- | :--- | :------------ | :------------ | | Total revenues | $2,419.2 | $2,096.0 | $1,846.3 | $323.2 | $249.7 | | Cost of sales | $514.9 | $483.7 | $470.5 | $31.2 | $13.2 | | Gross margin | 78.7% | 76.9% | 74.5% | 1.8% | 2.4% | - Cost of Sales increased in **2023** compared to **2022** primarily due to higher sales volumes[476](index=476&type=chunk) - Gross margin increased in **2023** primarily due to higher sales volume of products with higher margins, predominantly VOXZOGO, and lower per-unit manufacturing costs for enzyme products[476](index=476&type=chunk) - Gross margin is expected to increase modestly in future periods as the product mix shifts towards higher-margin commercial products[477](index=477&type=chunk) [Research and Development](index=62&type=section&id=Research%20and%20Development) Research and Development (R&D) expense increased by **$97.2 million** in **2023**, driven by higher spending in research and early pipeline activities, partially offset by decreased later-stage clinical program spending post-ROCTAVIAN approval R&D Expense by Category (in millions of U.S. Dollars) | Category | 2023 | 2022 | 2021 | 2023 vs. 2022 | 2022 vs. 2021 | | :------- | :--- | :--- | :--- | :------------ | :------------ | | Research and early pipeline | $393.1 | $313.9 | $275.9 | $79.2 | $38.0 | | Later-stage clinical programs | $62.6 | $119.0 | $244.4 | $(56.4) | $(125.4) | | Marketed Products | $291.1 | $216.7 | $108.5 | $74.4 | $108.2 | | **Total R&D expense** | **$746.8** | **$649.6** | **$628.8** | **$97.2** | **$20.8** | - The increase in R&D expense in **2023** was primarily due to higher spend in research and early pipeline activities, including pre-clinical studies for planned clinical trial application submissions[481](index=481&type=chunk) - Higher R&D spend on marketed products was partially offset by a decrease in later-stage clinical program spend due to the marketing approval of ROCTAVIAN in mid-2023[481](index=481&type=chunk) - R&D expense is expected to increase in future periods compared to **2023**, primarily due to higher spend on early pipeline and later-stage clinical programs[482](index=482&type=chunk) [Selling, General and Administrative](index=63&type=section&id=Selling,%20General%20and%20Administrative) Total Selling, General and Administrative (SG&A) expenses increased by **$83.3 million** in **2023**, primarily due to increased sales and marketing for ROCTAVIAN's launch, higher ERP system costs, an impairment charge, and unfavorable currency fluctuations SG&A Expenses (in millions of U.S. Dollars) | Category | 2023 | 2022 | 2021 | 2023 vs. 2022 | 2022 vs. 2021 | | :------- | :--- | :--- | :--- | :------------ | :------------ | | S&M expense | $488.4 | $450.3 | $405.1 | $38.1 | $45.2 | | G&A expense | $448.9 | $403.7 | $354.3 | $45.2 | $49.4 | | **Total SG&A expense** | **$937.3** | **$854.0** | **$759.4** | **$83.3** | **$94.6** | - The increase in Sales and Marketing (S&M) expense in **2023** was primarily a result of increased activities supporting the European and U.S. commercial launch of ROCTAVIAN[484](index=484&type=chunk) - The increase in General and Administrative (G&A) expense was primarily due to increased costs related to the ERP system and other strategic initiatives, an impairment charge in **2023** (for the first-generation VOXZOGO pen device), and unfavorable fluctuations of unhedged currencies[485](index=485&type=chunk) - SG&A expense is expected to increase in future periods due to the continued market expansion of commercial products and support for global business growth[487](index=487&type=chunk) [Intangible Asset Amortization and Contingent Consideration and Gain on Sale of Nonfinancial Assets](index=64&type=section&id=Intangible%20Asset%20Amortization%20and%20Contingent%20Consideration%20and%20Gain%20on%20Sale%20of%20Nonfinancial%20Assets) Amortization of intangible assets remained flat in **2023**, while changes in contingent consideration decreased due to milestone attainment, and the gain on sale of nonfinancial assets significantly decreased due to the absence of a PRV sale Intangible Asset Amortization, Contingent Consideration, and Gain on Sale (in millions of U.S. Dollars) | Metric | 2023 | 2022 | 2021 | 2023 vs. 2022 | 2022 vs. 2021 | | :----- | :--- | :--- | :--- | :------------ | :------------ | | Amortization of intangible assets | $62.2 | $62.8 | $61.9 | $(0.6) | $0.9 | | Changes in the fair value of contingent consideration | $— | $4.4 | $8.0 | $(4.4) | $(3.6) | | **Total intangible asset amortization and contingent consideration** | **$62.2** | **$67.2** | **$69.9** | **$(5.0)** | **$(2.7)** | | Gain on sale of nonfinancial assets | $— | $108.0 | $— | $(108.0) | $108.0 | - The decrease in changes in the fair value of contingent consideration in **2023** was attributable to the attainment of final commercial milestones in **2022**[489](index=489&type=chunk) - The decrease in Gain on Sale of Nonfinancial Assets in **2023** was due to the sale of a Priority Review Voucher (PRV) in **2022**, with no similar transaction in **2023**[489](index=489&type=chunk) [Interest Income](index=64&type=section&id=Interest%20Income) Interest income increased significantly by **$40.3 million** in **2023**, primarily due to higher money market and available-for-sale debt securities balances coupled with higher yields on the investment portfolio Interest Income (in millions of U.S. Dollars) | Metric | 2023 | 2022 | 2021 | 2023 vs. 2022 | 2022 vs. 2021 | | :----- | :--- | :--- | :--- | :------------ | :------------ | | Interest income | $58.3 | $18.0 | $10.5 | $40.3 | $7.5 | - The increase in Interest Income during **2023** compared to **2022** was primarily due to higher money market and available-for-sale debt securities balances and higher yields on the investment portfolio[491](index=491&type=chunk) - Interest Income is not expected to fluctuate significantly over the next **12 months** due to anticipated interest rates and yields on cash equivalents and investments[491](index=491&type=chunk) [Interest Expense](index=64&type=section&id=Interest%20Expense) Interest expense remained relatively flat in **2023**, primarily on convertible debt, and is expected to decrease over the next **12 months** due to the anticipated settlement of convertible debt maturing in **August 2024** Interest Expense (in millions of U.S. Dollars) | Metric | 2023 | 2022 | 2021 | 2023 vs. 2022 |

MOST PRODUCTIVE Continued significant revenue growth, intense focus on operational excellence, 6 Total Revenues of $646.2 million in the Fourth Quarter 2023 Increased 20% Q/Q Produced double-digit revenue growth and significant operating leverage in 2023, with non-GAAP diluted earnings per share increasing 36% Y/Y B:OMARIN (2) Non-GAAP Income is defined by the company as reported GAAP Net Income, excluding amortization expense, stock-based compensation expense, contingent consideration, and, in certain peri ...

Financial Data and Key Metrics Changes - Total revenue grew 20% in Q4 2023 compared to Q4 2022, and 15% for the full year 2023 compared to the prior year, with constant currency growth of 25% in Q4 and 20% for the full year [11][12] - Non-GAAP earnings per share increased 48% in Q4 versus Q4 2022, and 36% year-over-year for the full year [12][48] - GAAP net income for Q4 was $20.4 million, contributing to $168 million for the full year, with GAAP diluted earnings per share of $0.87 [47][48] Business Line Data and Key Metrics Changes - VOXZOGO revenues reached $146 million in Q4, representing 118% quarter-over-quarter growth, with a year-over-year growth of 178% [13][44] - Enzyme products generated over $1.7 billion in net product revenues for the full year 2023, an increase of 4% year-over-year [43] - Double-digit revenue growth was observed in both VIMIZIM and PALYNZIQ in Q4, partially offset by lower KUVAN revenues [44] Market Data and Key Metrics Changes - 70% of new US prescriptions for VOXZOGO in Q4 were for children under five, following FDA's age expansion approval [14] - The company is actively engaging global health authorities regarding development programs in idiopathic short stature and multiple genetic short stature pathway conditions [19] Company Strategy and Development Direction - The company aims to maximize the VOXZOGO opportunity and expand into multiple other statural conditions [20] - A strategic portfolio review is underway to prioritize R&D assets with the highest potential patient impact and value creation for shareholders [25][30] - The company plans to increase profitability faster than originally planned, with guidance reflecting double-digit revenue growth for 2024 [26][55] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for VOXZOGO and the potential for ROCTAVIAN, with expectations for patient treatment in 2024 [21][24] - The company is focused on disciplined spending and prioritization of impactful R&D assets, with updates to be communicated at an Investor Day later in 2024 [25][37] - Management noted the importance of aligning patient motivation, payer support, and treatment site readiness for successful ROCTAVIAN treatment [23] Other Important Information - The company is transitioning to a cash flow positive status and is committed to developing a capital allocation strategy that maximizes shareholder value [123] - Non-GAAP operating margin for 2024 is guided to be between 23% and 24%, representing a 4 percentage point expansion from 2023 [55] Q&A Session Summary Question: EPS guidance for 2024 and assumptions - Management indicated that the non-GAAP EPS guidance midpoint implies 30% growth over 2023, with some tax benefits and unique SG&A charges impacting the analysis [60][62] Question: R&D portfolio prioritization criteria - The strategic assessment will rank and prioritize assets based on time, cost, market opportunity, and competitive landscape [65][66] Question: Thoughts on divesting gene therapy platform - Management is still assessing ROCTAVIAN's opportunity and will evaluate gene therapy exposure in the pipeline [71][72] Question: VOXZOGO revenue dynamics and channel inventory - Management clarified that Q4 revenue included additional commercial supply, but Q1 growth may not replicate the same dynamic [74] Question: Update on VOXZOGO's approval status - No specific updates on regulatory status, but management emphasized the sustained effect of VOXZOGO as a key feature [84] Question: Commercial investments impact on VOXZOGO - Management noted good returns on commercial investments, with a focus on expanding the prescriber base [90] Question: Market opportunity for VOXZOGO - Management indicated that approximately 20,000 patients are eligible for VOXZOGO across 41 active markets [118] Question: Future capital allocation strategy - Management is committed to internal innovation while also reviewing the pipeline for potential cuts and reinvestments [123]

BioMarin Pharmaceutical (BMRN) reported $646.21 million in revenue for the quarter ended December 2023, representing a year-over-year increase of 20.2%. EPS of $0.49 for the same period compares to $0.36 a year ago.The reported revenue compares to the Zacks Consensus Estimate of $636.71 million, representing a surprise of +1.49%. The company delivered an EPS surprise of +11.36%, with the consensus EPS estimate being $0.44.While investors scrutinize revenue and earnings changes year-over-year and how they co ...

BioMarin Pharmaceutical (BMRN) came out with quarterly earnings of $0.49 per share, beating the Zacks Consensus Estimate of $0.44 per share. This compares to earnings of $0.36 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 11.36%. A quarter ago, it was expected that this rare disease biopharmaceutical would post earnings of $0.46 per share when it actually produced earnings of $0.46, delivering no surprise.Over the last four ...

Exhibit 99.1 BioMarin Reports Record Financial Results for the Fourth Quarter and Full-year 2023 and Provides Financial Guidance for 2024 SAN RAFAEL, Calif., February 22, 2024 – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the fourth quarter and full year ended December 31, 2023. 1 | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | |----------------------------------------------|-------|----------------|-------------|------------------------------------- ...