Biomarin Pharmaceutical, Inc. (NASDAQ:BMRN) Q1 2023 Earnings Conference Call April 26, 2023 4:30 PM ET Company Participants Traci McCarty - Group VP, IR Jean-Jacques Bienaime - Chairman & CEO Jeffrey Ajer - Chief Commercial Officer Henry Fuchs - President, Worldwide Research & Development Brian Mueller - CFO & EVP Conference Call Participants Salveen Richter - Goldman Sachs Group Geoffrey Meacham - Bank of America Merrill Lynch Christopher Raymond - Piper Sandler & Co. Timothy Lugo - William Blair & Company ...

Financial Data and Key Metrics Changes - BioMarin achieved total revenues of $2.1 billion for the full year 2022, marking a record financial achievement for the company [33] - The company reported a revenue growth of close to 20% in Q4 2022, with total revenues of $538 million [34] - GAAP net income and non-GAAP income growth were achieved despite recognizing approximately $23 million in charges to SG&A expenses in 2022, primarily in Q4 [27] Business Line Data and Key Metrics Changes - VOXZOGO generated full-year global revenue of $169 million in 2022, with guidance for 2023 set between $330 million and $380 million, indicating over 100% growth from the midpoint compared to 2022 [12] - PALYNZIQ therapy in the U.S. and Europe saw net product revenue growth of 7% in 2022, totaling $255 million [15] - ROCTAVIAN is projected to generate revenues between $100 million and $200 million in 2023, contingent on U.S. approval [38] Market Data and Key Metrics Changes - As of the end of Q4 2022, only 6% of the total addressable patient population had received VOXZOGO treatment, indicating significant growth potential [12] - In Germany, a major payer agreement has been established for ROCTAVIAN, although the commercial ramp has been hindered by lengthy negotiations for additional outcomes-based agreements [39] Company Strategy and Development Direction - The company aims for over 15% top-line growth in 2023, with a focus on sustainable profitability and leveraging its enzyme products alongside new market opportunities from VOXZOGO and ROCTAVIAN [33][43] - BioMarin is actively pursuing regulatory approvals to expand VOXZOGO's label to younger patients, which could significantly increase the addressable market [13][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the interest from prescribers and patients for ROCTAVIAN, despite the lengthy reimbursement process in Germany [32] - The company anticipates increased demand for all brands in 2023, including enzyme products, VOXZOGO, and ROCTAVIAN, with a commitment to growth and sustainable profitability [43] Other Important Information - The company is transitioning to a new methodology for calculating non-GAAP income to better align with its profitable business model and peer group [75] - BioMarin is investing in its largest early-stage research pipeline to fuel growth throughout the decade [74] Q&A Session Summary Question: What is the expected timeline for revenue flow from ROCTAVIAN in Germany? - Management confirmed that the 10 patients undergoing companion diagnostic testing are in Germany and emphasized the importance of these tests as a step towards treatment [80][81] Question: What is the status of the three-year data submission for ROCTAVIAN? - The company indicated that the assessment of whether the submission constitutes a major amendment is subjective and cannot provide specific guidance at this time [85] Question: How many U.S. treatment centers are expected to be ready for ROCTAVIAN at launch? - The company plans to target a small number of the largest and most capable hemophilia treatment centers in the U.S. for the launch of ROCTAVIAN [96] Question: What are the implications of the outcomes-based agreements in Germany? - Management explained that while these agreements will be superseded by federal reimbursement, they still provide value in facilitating patient access to therapy [89][116]

Part I [Item 1. Business](index=5&type=section&id=Item%201.%20Business) BioMarin Pharmaceutical Inc. is a global biotechnology company focused on developing and commercializing targeted therapies for rare genetic disorders, reporting nearly **$2.1 billion** in 2022 revenues - **BioMarin** is a global biotechnology company founded in 1997, specializing in **targeted therapies for rare genetic conditions** through **genetic discovery** and a diverse pipeline of commercial, clinical, and preclinical candidates[115](index=115&type=chunk)[774](index=774&type=chunk)[1111](index=1111&type=chunk) 2022 Total Revenues | Metric | Amount (Millions USD) | | :----------- | :-------------------- | | Total Revenues | 2,096.0 | - Key business developments in 2022 include the conditional approval of **ROCTAVIAN** in the EU for severe hemophilia A, positive three-year data from **ROCTAVIAN**'s Phase 3 study, FDA acceptance of **ROCTAVIAN**'s BLA resubmission, and **VOXZOGO** approvals in Australia and Japan for achondroplasia[137](index=137&type=chunk)[775](index=775&type=chunk)[961](index=961&type=chunk) [Overview](index=5&type=section&id=Overview) The company is a global biotechnology firm specializing in **targeted therapies for rare genetic conditions** through **genetic discovery** and a diverse pipeline - **BioMarin Pharmaceutical Inc.** is a global biotechnology company focused on **genetic discovery** to develop and commercialize **targeted therapies for rare genetic conditions**, with a diverse pipeline of innovative commercial, clinical, and preclinical candidates[115](index=115&type=chunk)[774](index=774&type=chunk)[1111](index=1111&type=chunk) [Recent Developments](index=5&type=section&id=Recent%20Developments) **BioMarin** achieved nearly **$2.1 billion** in 2022 revenues, driven by **VOXZOGO**'s launch and key pipeline advancements including **ROCTAVIAN**'s EU approval - In 2022, **BioMarin** achieved nearly **$2.1 billion** in total revenues, with significant contributions from **VOXZOGO**'s launch and key advancements in its product development pipeline[137](index=137&type=chunk)[775](index=775&type=chunk) - Notable developments include the conditional approval of **ROCTAVIAN** in the EU for severe hemophilia A, positive three-year data from its Phase 3 study, FDA acceptance of **ROCTAVIAN**'s BLA resubmission, and **VOXZOGO** approvals in Australia and Japan for achondroplasia[137](index=137&type=chunk)[775](index=775&type=chunk) [Commercial Products](index=5&type=section&id=Commercial%20Products) This section details **BioMarin**'s commercial product portfolio, including 2022 revenues, regulatory status, and distribution specifics Commercial Products and 2022 Revenues | Product Name | Indication | 2022 Revenues (Millions USD) | | :----------------------- | :------------------ | :--------------------------- | | VIMIZIM (elosulfase alpha) | MPS IVA | 663.8 | | NAGLAZYME (galsulfase) | MPS VI | 443.8 | | PALYNZIQ (pegvaliase-pqpz) | PKU | 255.0 | | KUVAN (sapropterin dihydrochloride) | PKU | 227.6 | | VOXZOGO (vosoritide) | Achondroplasia | 169.1 | | BRINEURA (cerliponase alfa) | CLN2 | 154.3 | | ROCTAVIAN (valoctocogene roxaparvovec) | Severe Hemophilia A | — | | ALDURAZYME (laronidase) | MPS I | 128.4 | - **ROCTAVIAN** received conditional approval in the EU in Q3 2022, with no sales in 2022, and is undergoing FDA review with a PDUFA target action date of March 31, 2023[138](index=138&type=chunk)[151](index=151&type=chunk)[598](index=598&type=chunk) - **VOXZOGO** is approved for achondroplasia in various age ranges globally, including ages five and older in the U.S. and two and older in the EU/Australia, and all ages in Japan[138](index=138&type=chunk)[146](index=146&type=chunk)[598](index=598&type=chunk) - **PALYNZIQ** is distributed in the U.S. through a REMS program to mitigate anaphylaxis risk, requiring certified prescribers, pharmacies, and patient education, along with epinephrine availability[142](index=142&type=chunk)[168](index=168&type=chunk)[169](index=169&type=chunk) [Clinical Development Programs](index=9&type=section&id=Clinical%20Development%20Programs) The company's clinical pipeline includes **ROCTAVIAN**, **BMN 255** for hyperoxaluria, and **BMN 331** for Hereditary Angioedema Clinical Development Programs as of December 31, 2022 | Product Candidate | Target Indication | Stage | | :---------------- | :------------------------ | :-------------------- | | ROCTAVIAN | Severe Hemophilia A | FDA regulatory review | | BMN 255 | Hyperoxaluria | Clinical Phase 1/2 | | BMN 331 | Hereditary Angioedema (HAE) | Clinical Phase 1/2 | - **BMN 255**, a small-molecule therapy for hyperoxaluria in chronic liver disease, showed a rapid and potent increase in plasma glycolate in its Phase 1/2 study, with an expanded study planned for 2023[156](index=156&type=chunk)[214](index=214&type=chunk)[800](index=800&type=chunk) - **BMN 331**, a **gene therapy** for Hereditary Angioedema (HAE), is in Phase 1/2, with the first participant at the 6e13vg/kg dose demonstrating C1-Inhibitor levels approaching the normal range[157](index=157&type=chunk)[186](index=186&type=chunk)[800](index=800&type=chunk) [Manufacturing](index=9&type=section&id=Manufacturing) **BioMarin** manufactures APIs in California and Ireland, adhering to **current Good Manufacturing Practices (cGMPs)**, with a dedicated **gene therapy** facility for **ROCTAVIAN** - **BioMarin** manufactures active pharmaceutical ingredients (API) for **ALDURAZYME**, **NAGLAZYME**, **PALYNZIQ**, and **VOXZOGO** in Novato, California, and for **BRINEURA** and **VIMIZIM** in Shanbally, Cork, Ireland[187](index=187&type=chunk)[215](index=215&type=chunk) - The company's facilities and those of third-party manufacturers are subject to periodic inspections to ensure compliance with **current Good Manufacturing Practices (cGMPs)** by regulatory authorities like the FDA and EC[158](index=158&type=chunk)[187](index=187&type=chunk)[215](index=215&type=chunk) - A commercial-scale **gene therapy** manufacturing facility in Novato, California, commissioned in July 2017, supports **cGMP** production of **ROCTAVIAN** for commercial sales in Europe and clinical development activities for **BMN 331**[159](index=159&type=chunk) [Raw Materials](index=10&type=section&id=Raw%20Materials) Raw materials are sourced from single suppliers, with risks mitigated through inventory and multiple sourcing strategies - Raw materials and supplies for product production are sourced from single or sole suppliers in some instances, with policies adopted to minimize supply risks through inventory maintenance and multiple sourcing strategies for critical materials[189](index=189&type=chunk)[217](index=217&type=chunk) [Sales and Marketing](index=10&type=section&id=Sales%20and%20Marketing) **BioMarin** markets products directly and through distributors, utilizing third-party logistics and vendors for support - **BioMarin** markets its products (excluding **ALDURAZYME**) directly through commercial teams in the U.S., Europe, South America, and other key markets, and through distributors in other selected markets[161](index=161&type=chunk)[218](index=218&type=chunk) - The company utilizes third-party logistics for storage and distribution and third-party vendors for advertising, market research, and sales support[219](index=219&type=chunk) [Customers](index=10&type=section&id=Customers) Customers include specialty pharmacies and hospitals, with **three customers generating 37% of net product revenue** in 2022 - Customers for **BioMarin**'s products (excluding **ALDURAZYME**) include specialty pharmacies, hospitals, and non-U.S. government agencies, with sales also made to authorized distributors and large pharmaceutical wholesalers[88](index=88&type=chunk)[191](index=191&type=chunk) - In 2022, **three customers generated 37% of net product revenue**, and Sanofi is the sole customer for **ALDURAZYME**, responsible for its global marketing and sales[192](index=192&type=chunk)[844](index=844&type=chunk) [Competition](index=11&type=section&id=Competition) The biopharmaceutical industry is highly competitive, with **KUVAN** facing generic competition and other products facing various rivals - The biopharmaceutical industry is highly competitive, with numerous public and private companies, universities, and non-profit organizations developing treatments for the same indications as **BioMarin**'s products and candidates[193](index=193&type=chunk)[221](index=221&type=chunk) - **KUVAN** faces generic competition globally, including multiple generic versions in the U.S. and EU, which has adversely affected and is expected to continue affecting its revenues[89](index=89&type=chunk)[172](index=172&type=chunk)[221](index=221&type=chunk) - **ROCTAVIAN**, a **gene therapy** for severe hemophilia A, faces competition from marketed recombinant factor VIII therapies, a novel bispecific antibody, and clinical-stage **gene therapy** and non-factor replacement candidates[226](index=226&type=chunk) - **VOXZOGO** for achondroplasia, **BMN 255** for hyperoxaluria, and **BMN 331** for HAE also face potential competition from various clinical and preclinical stage products from other companies[196](index=196&type=chunk)[198](index=198&type=chunk)[199](index=199&type=chunk)[224](index=224&type=chunk)[227](index=227&type=chunk) [Patents, Proprietary Rights and Regulatory Exclusivity](index=12&type=section&id=Patents%2C%20Proprietary%20Rights%20and%20Regulatory%20Exclusivity) **BioMarin** relies on its **intellectual property**, including patents and **regulatory exclusivity**, to protect its revenue streams - **BioMarin**'s success relies on its **intellectual property** portfolio, which includes patents, trade secrets, and trademarks, to support revenue streams and create barriers to competitors[228](index=228&type=chunk) - U.S. and most foreign patents are generally effective for 20 years from the earliest filing date, with potential extensions for regulatory review periods (up to five years in the U.S. and EU)[200](index=200&type=chunk) Regulatory Exclusivity for Commercial Products | Commercial Products | United States Orphan Drug Exclusivity Expiration | United States Biologic Exclusivity Expiration | European Union Orphan Drug Exclusivity Expiration | | :------------------ | :----------------------------------------------- | :-------------------------------------------- | :------------------------------------------------ | | BRINEURA | 2024 | 2029 | 2027 | | PALYNZIQ | 2025 | 2030 | 2029 | | ROCTAVIAN | Pending | Pending | 2032 | | VIMIZIM | Expired | 2026 | 2024 | | VOXZOGO | 2028 | Not Applicable | 2031 | [Government Regulation](index=14&type=section&id=Government%20Regulation) **BioMarin**'s products are subject to extensive government regulations covering development, manufacturing, and commercialization - **BioMarin**'s products are subject to extensive federal, state, local, and non-U.S. regulations governing development, manufacture, commercialization, pricing, and reimbursement, requiring approvals from agencies like the FDA and EMA[205](index=205&type=chunk)[233](index=233&type=chunk) [Human Capital](index=24&type=section&id=Human%20Capital) **BioMarin** had **3,082 global employees** in 2022, undergoing a reorganization, and committed to **diversity, equity, and inclusion (DEI)** Global Full-Time Employees as of December 31, 2022 | Department | Number of Employees | | :--------------------- | :------------------ | | Operations | 1,343 | | Research & Development | 736 | | Sales & Marketing | 495 | | Administration | 508 | | **Total Worldwide** | **3,082** | | U.S. and Canada | 2,066 | | Other non-U.S. | 1,016 | - In October 2022, **BioMarin** announced a reorganization plan, including a reduction of approximately **120 employees** (**4% of its global workforce**), primarily from U.S. operations, incurring **$23.0 million** in severance and termination benefits[565](index=565&type=chunk)[617](index=617&type=chunk)[964](index=964&type=chunk) - As of December 31, 2022, **47% of U.S. employees** were racial and ethnic minorities, and **50% of the global workforce** and **47% of director-level and above positions** were held by women, reflecting the company's commitment to **diversity, equity, and inclusion (DEI)**[330](index=330&type=chunk) [Other Information](index=26&type=section&id=Other%20Information) **BioMarin** was incorporated in 1996, with its SEC filings and annual reports available on its website and the SEC's - **BioMarin Pharmaceutical Inc.** was incorporated in Delaware in October 1996, with principal executive offices in San Rafael, California[305](index=305&type=chunk)[335](index=335&type=chunk) - Annual reports on Form 10-K, quarterly reports on Form 10-Q, proxy statements, current reports on Form 8-K, and amendments are available free of charge on the company's website (www.bmrn.com) and the SEC's website (www.sec.gov)[305](index=305&type=chunk)[335](index=335&type=chunk) [Item 1A. Risk Factors](index=27&type=section&id=Item%201A.%20Risk%20Factors) Investing in **BioMarin**'s securities involves significant risks due to the dynamic and rapidly changing biopharmaceutical industry, including challenges in product coverage, intense competition, and regulatory uncertainties - An investment in **BioMarin**'s securities involves a **high degree of risk** due to the dynamic and rapidly changing biopharmaceutical industry[306](index=306&type=chunk)[337](index=337&type=chunk) - The company faces risks related to obtaining and maintaining adequate coverage and reimbursement for its expensive products, especially given small target patient populations requiring high per-patient prices for profitability[307](index=307&type=chunk)[338](index=338&type=chunk)[341](index=341&type=chunk) - Generic versions of **KUVAN** are adversely affecting and will continue to affect revenues, with potential for faster decline if patent challenges are successful or new generic versions are introduced[346](index=346&type=chunk)[367](index=367&type=chunk) [Business and Operational Risks](index=27&type=section&id=Business%20and%20Operational%20Risks) Profitability relies on market share and high prices, with risks from changing treatments and failure to expand the pipeline - The company's profitability depends on achieving significant market share and maintaining high per-patient prices for its products, especially for those targeting small patient populations like **BRINEURA**, **NAGLAZYME**, and **VIMIZIM**[341](index=341&type=chunk)[342](index=342&type=chunk) - Changes in disease treatment methods, such as the widespread adoption of **gene therapy** over enzyme replacement therapy, could reduce demand for **BioMarin**'s products and adversely affect revenues[313](index=313&type=chunk)[343](index=343&type=chunk) - Failure to successfully develop new products, or compete effectively for acquisitions, joint ventures, and licensing opportunities, could impair the expansion of **BioMarin**'s product pipeline and overall growth[314](index=314&type=chunk)[365](index=365&type=chunk)[366](index=366&type=chunk) [Regulatory Risks](index=29&type=section&id=Regulatory%20Risks) Regulatory approvals are lengthy and uncertain, with ongoing compliance risks, potential price controls, and **orphan drug exclusivity** challenges - Obtaining regulatory approval for product candidates is a lengthy, expensive, and uncertain process, with no guarantee of approval or that approval in one region ensures approval in others[348](index=348&type=chunk)[370](index=370&type=chunk) - Approved products are subject to extensive ongoing regulatory requirements, and non-compliance or unanticipated problems can lead to penalties, restrictions, or withdrawal of products from the market[354](index=354&type=chunk)[376](index=376&type=chunk)[380](index=380&type=chunk) - Government price controls, healthcare reforms (like the IRA and PPACA), and cost-containment measures in the U.S. and internationally could restrict product pricing, reduce reimbursement, and adversely affect revenues and profitability[360](index=360&type=chunk)[413](index=413&type=chunk)[416](index=416&type=chunk) - Failure to obtain or maintain **orphan drug exclusivity** for eligible products could lead to competition from similar drugs, reducing revenues, especially for products with limited patent protection[389](index=389&type=chunk)[417](index=417&type=chunk) [Risks Related to Our Gene Therapy Programs](index=36&type=section&id=Risks%20Related%20to%20Our%20Gene%20Therapy%20Programs) **Gene therapies** like **ROCTAVIAN** face unique development, manufacturing, regulatory, and commercialization risks, including data access limitations - **Gene therapy** products, like **ROCTAVIAN** and **BMN 331**, are based on novel technology, presenting unique development, manufacturing, regulatory, and treatment risks, including uncertainty about durability of effect, potential for side effects, and manufacturing complexities[394](index=394&type=chunk)[395](index=395&type=chunk)[396](index=396&type=chunk)[423](index=423&type=chunk)[425](index=425&type=chunk)[426](index=426&type=chunk) - Commercialization of **gene therapies** faces uncertainty regarding pricing, coverage, and reimbursement due to their novelty and high cost, with **BioMarin** entering outcomes-based agreements for **ROCTAVIAN** that involve potential repayments[397](index=397&type=chunk)[398](index=398&type=chunk)[427](index=427&type=chunk) - Data access plans for **ROCTAVIAN**'s main clinical trials restrict management's review of emerging efficacy data, which could adversely impact trial outcomes as adjustments cannot be made based on real-time results[401](index=401&type=chunk)[430](index=430&type=chunk) [Financial and Financing Risks](index=38&type=section&id=Financial%20and%20Financing%20Risks) **BioMarin**'s profitability is uncertain, requiring substantial capital for operations and debt repayment, with significant indebtedness impacting flexibility - **BioMarin** has historically operated at a **net loss** and its future profitability is uncertain, requiring substantial capital for operations, product development, and debt repayment[402](index=402&type=chunk)[458](index=458&type=chunk) Cash, Cash Equivalents, and Investments (as of December 31, 2022) | Category | Amount (Billions USD) | | :------------------------------------- | :-------------------- | | Cash, cash equivalents and investments | 1.6 | | Debt obligations (undiscounted) | 1.1 | - The company's substantial indebtedness, including **convertible notes** due in 2024 and 2027, may limit its financial flexibility, access to capital, and increase borrowing costs, potentially impacting operations and financial results[408](index=408&type=chunk)[434](index=434&type=chunk)[462](index=462&type=chunk) [Manufacturing Risks](index=40&type=section&id=Manufacturing%20Risks) Manufacturing risks include **current Good Manufacturing Practices (cGMPs)** non-compliance, production complexities, reliance on single suppliers, and potential termination of the Sanofi agreement - Failure to comply with manufacturing regulations (**cGMPs**) by **BioMarin** or its third-party manufacturers could lead to delayed approvals, fines, product recalls, or suspension of production, adversely affecting financial results[438](index=438&type=chunk)[439](index=439&type=chunk)[466](index=466&type=chunk) - The complexity of manufacturing processes for products and candidates means the company may be unable to produce sufficient quantities at acceptable costs, potentially delaying or terminating development programs or impacting commercial demand and margins[440](index=440&type=chunk)[467](index=467&type=chunk)[468](index=468&type=chunk) - Reliance on single-source suppliers for critical raw materials and a limited number of manufacturing facilities creates risks of supply interruptions due to various factors, which could disrupt inventory and reduce revenues[445](index=445&type=chunk)[472](index=472&type=chunk)[473](index=473&type=chunk) - Termination of the Manufacturing, Marketing and Sales Agreement with Sanofi for **ALDURAZYME** could prevent **BioMarin** from commercializing the product or significantly delay/diminish its ability to do so, with potential financial implications[449](index=449&type=chunk)[450](index=450&type=chunk)[474](index=474&type=chunk)[476](index=476&type=chunk) [Risks Related to International Operations](index=42&type=section&id=Risks%20Related%20to%20International%20Operations) International operations face risks from complexity, **intellectual property** protection, tax changes, political instability, currency fluctuations, and export controls - Significant international sales and operations expose **BioMarin** to risks including increased complexity and costs, difficulty in management, diminished **intellectual property** protection, negative tax law changes, and political/economic instability (e.g., Russia's invasion of Ukraine)[451](index=451&type=chunk)[477](index=477&type=chunk)[478](index=478&type=chunk)[505](index=505&type=chunk) - Reliance on special access programs for initial international sales of newly launched products (e.g., **VOXZOGO**) poses risks, as changes or elimination of these programs could adversely affect product sales and revenues[454](index=454&type=chunk)[480](index=480&type=chunk)[507](index=507&type=chunk) - Exposure to foreign currency exchange rate fluctuations, particularly the Euro, can impact revenues and expenses, with a stronger USD adversely affecting results despite hedging strategies[481](index=481&type=chunk)[508](index=508&type=chunk)[509](index=509&type=chunk) - Compliance with U.S. export control laws and economic sanctions (e.g., against Russia and Belarus) is critical; non-compliance or changes in these laws could hinder sales, operations, and lead to substantial penalties[6](index=6&type=chunk)[456](index=456&type=chunk)[482](index=482&type=chunk)[510](index=510&type=chunk) [Intellectual Property Risks](index=45&type=section&id=Intellectual%20Property%20Risks) IP risks include patent uncertainty, trade secret protection challenges, potential infringement claims, and public disclosure of proprietary data - The **patent positions** of biopharmaceutical products are complex and uncertain, especially for products with publicly available key information, potentially preventing **BioMarin** from obtaining or enforcing strong patent protection[10](index=10&type=chunk)[486](index=486&type=chunk)[514](index=514&type=chunk) - Trade secrets may not be adequately protected, with risks of unintentional or willful disclosure by employees/contractors and the expense/difficulty of enforcing claims, especially outside the U.S[11](index=11&type=chunk) - Competitors may claim infringement of their **intellectual property**, leading to lawsuits, substantial damages, injunctions, or the need for expensive and potentially unavailable licenses, which could adversely affect business[490](index=490&type=chunk)[517](index=517&type=chunk)[518](index=518&type=chunk) - Public disclosure of clinical trial data and proprietary information by regulatory authorities (e.g., EMA) could adversely affect **BioMarin**'s business by revealing confidential methodologies and enabling competitors[515](index=515&type=chunk)[516](index=516&type=chunk) [Risks Related to Ownership of Our Securities](index=46&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Securities) **Stock price volatility**, dilution from **convertible notes**, and **Anti-takeover provisions** pose risks to security ownership - **BioMarin**'s **stock price has been and may remain volatile**, influenced by factors such as product sales, generic competition, regulatory actions, patent disputes, negative publicity, and broad market fluctuations[492](index=492&type=chunk)[493](index=493&type=chunk)[520](index=520&type=chunk)[521](index=521&type=chunk) - Conversion of the company's **convertible notes** will dilute existing stockholders' ownership interests and could depress the common stock price, potentially encouraging short selling[19](index=19&type=chunk)[494](index=494&type=chunk) - **Anti-takeover provisions** in Delaware law and **BioMarin**'s charter documents, such as restrictions on calling stockholder meetings and the board's authority to issue preferred stock, may make a change in control more difficult[21](index=21&type=chunk)[496](index=496&type=chunk) [General Risk Factors](index=49&type=section&id=General%20Risk%20Factors) General risks include talent retention, **tax laws and regulations** changes, **healthcare laws** and **data privacy laws** compliance failures, **information technology systems** disruptions, natural disasters, and macroeconomic impacts - **BioMarin**'s success depends heavily on its ability to attract and retain qualified scientific, technical, and managerial personnel, with intense competition for talent potentially harming business operations if recruitment and retention efforts are unsuccessful[499](index=499&type=chunk)[527](index=527&type=chunk) - New or modified **tax laws and regulations**, including international initiatives like BEPS 2.0, could adversely affect **BioMarin**'s business and financial condition by increasing tax expenses or creating tax uncertainty[51](index=51&type=chunk)[500](index=500&type=chunk)[528](index=528&type=chunk)[529](index=529&type=chunk) - Non-compliance with **healthcare laws** (e.g., Anti-Kickback Statute, False Claims Act) and **data privacy laws** (e.g., GDPR, CCPA, CPRA) could result in significant penalties, regulatory scrutiny, and reputational harm[501](index=501&type=chunk)[502](index=502&type=chunk)[503](index=503&type=chunk)[530](index=530&type=chunk)[532](index=532&type=chunk) - Significant reliance on **information technology systems** means any failure, inadequacy, interruption, or **cybersecurity incident** could harm business operations, reputation, financial condition, and results[31](index=31&type=chunk)[535](index=535&type=chunk) - Natural disasters (e.g., earthquakes, fires) or other unforeseen events could damage facilities or those of third-party manufacturers/suppliers, impairing production and commercialization efforts[34](index=34&type=chunk)[538](index=538&type=chunk) - Macroeconomic factors like inflation, interest rates, foreign currency exchange rates, and global public health threats (e.g., COVID-19) could adversely affect business, financial results, and the ability of customers/distributors to purchase products[35](index=35&type=chunk)[539](index=539&type=chunk)[541](index=541&type=chunk)[869](index=869&type=chunk) [Item 1B. Unresolved Staff Comments](index=55&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) As of the reporting date, there are no unresolved staff comments from the SEC - There are no unresolved staff comments as of the reporting date[542](index=542&type=chunk) [Item 2. Properties](index=55&type=section&id=Item%202.%20Properties) **BioMarin**'s significant owned and leased properties as of December 31, 2022, include corporate headquarters, manufacturing, laboratory, and office facilities in California and Ireland, which are deemed adequate for foreseeable operations Significant Owned and Leased Properties (as of December 31, 2022) | 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 | 164,000 | Office and warehouse | 2023 | | Shanbally facility, Cork, Ireland | 260,700 | Manufacturing, laboratory and office | Owned property | - These properties, along with other smaller leased office facilities globally, are expected to be adequate for **BioMarin**'s operations for the foreseeable future[66](index=66&type=chunk) [Item 3. Legal Proceedings](index=55&type=section&id=Item%203.%20Legal%20Proceedings) **BioMarin** is involved in several legal proceedings, including shareholder class actions regarding **ROCTAVIAN** and **BMN 307**, and a derivative action related to **ROCTAVIAN** - A shareholder class action lawsuit was filed in September 2020, alleging **BioMarin** made materially false or misleading statements regarding **ROCTAVIAN**'s clinical trials and BLA, specifically concerning the durability of effect and FDA approval without additional data[544](index=544&type=chunk) - In October 2021, another purported securities class action was filed, alleging false or misleading statements about **BMN 307**'s safety profile and overstating its clinical and commercial prospects; this complaint was dismissed with prejudice in February 2023, but plaintiffs plan to appeal[67](index=67&type=chunk) - In January 2023, a shareholder derivative action was filed against certain officers and directors, asserting breach of fiduciary duty claims arising from the facts underlying the **ROCTAVIAN** securities class action[39](index=39&type=chunk) - **BioMarin** believes all claims in these lawsuits have no merit and intends to vigorously defend these actions[39](index=39&type=chunk)[67](index=67&type=chunk)[544](index=544&type=chunk) [Item 4. Mine Safety Disclosures](index=56&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) The company has no disclosures to report regarding mine safety - Not applicable[546](index=546&type=chunk) Part II [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=57&type=section&id=Item%205.%20Market%20for%20Registrant's%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) **BioMarin**'s common stock is listed on the **Nasdaq Global Select Market** under "BMRN", with **186,528,784 outstanding shares** held by **35 holders of record** as of February 16, 2023 - **BioMarin**'s common stock is listed under the symbol "BMRN" on the **Nasdaq Global Select Market**[42](index=42&type=chunk)[70](index=70&type=chunk) - As of February 16, 2023, there were **35 holders of record** of **186,528,784 outstanding shares** of common stock[549](index=549&type=chunk) - The company has never paid cash dividends on its common stock and does not anticipate paying them in the foreseeable future[548](index=548&type=chunk) - No unregistered securities were sold during the year ended December 31, 2022, and there were no issuer purchases of equity securities[43](index=43&type=chunk)[70](index=70&type=chunk)[549](index=549&type=chunk) Comparative Stock Performance (Indexed to $100) | Index | 2017 | 2018 | 2019 | 2020 | 2021 | 2022 | | :--------------------------- | :----- | :----- | :----- | :----- | :----- | :----- | | BioMarin Pharmaceutical Inc. | $100.00 | $95.49 | $94.82 | $98.34 | $99.08 | $116.06 | | Nasdaq Composite Index | $100.00 | $97.16 | $132.81 | $192.47 | $235.15 | $158.65 | | Nasdaq Biotechnology | $100.00 | $91.14 | $114.02 | $144.15 | $144.18 | $129.59 | | SPDR S&P Biotech ETF | $100.00 | $84.72 | $112.31 | $166.59 | $132.53 | $98.25 | [Item 6. [Reserved]](index=58&type=section&id=Item%206.%20%5BReserved%5D) This item is reserved and contains no information - This item is reserved[73](index=73&type=chunk) [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=59&type=section&id=Item%207.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides an overview of **BioMarin**'s financial performance, liquidity, and capital resources, highlighting 2022 financial results, business developments, and critical accounting estimates - **BioMarin** is a global biotechnology company focused on developing and commercializing **targeted therapies for rare genetic conditions**, with a diverse pipeline of commercial, clinical, and preclinical candidates[59](index=59&type=chunk)[774](index=774&type=chunk)[958](index=958&type=chunk) Key Financial Highlights (Millions USD) | Metric | 2022 | 2021 | 2020 | | :----------------------------------- | :-------- | :-------- | :-------- | | Total revenues | $2,096.0 | $1,846.3 | $1,860.5 | | Cost of sales | $483.7 | $470.5 | $524.3 | | Research and development (R&D) expense | $649.6 | $628.8 | $628.1 | | Selling, general and administrative (SG&A) expense | $854.0 | $759.4 | $737.7 | | Gain on sale of nonfinancial assets, net | $(108.0) | — | $(52.8) | | Provision for (benefit from) income taxes | $8.0 | $(11.3) | $(903.0) | | Net income (loss) | $141.6 | $(64.1) | $854.0 | - The company expects continued growth in Net Product Revenues, excluding **KUVAN** due to generic competition, and plans to invest in its R&D pipeline and commercial infrastructure for 2023[566](index=566&type=chunk)[965](index=965&type=chunk) [Overview](index=59&type=section&id=Overview) **BioMarin** is a global biotechnology company focused on developing and commercializing **targeted therapies for rare genetic conditions** - **BioMarin** is a global biotechnology company focused on **genetic discovery** to develop and commercialize **targeted therapies for rare genetic conditions**, with a diverse pipeline of innovative commercial, clinical, and preclinical candidates[59](index=59&type=chunk)[774](index=774&type=chunk)[937](index=937&type=chunk) [2022 Financial Highlights](index=60&type=section&id=2022%20Financial%20Highlights) This section presents key financial metrics for 2022, 2021, and 2020, including revenues, expenses, and net income Key Financial Highlights (Millions USD) | Metric | 2022 | 2021 | 2020 | | :----------------------------------- | :-------- | :-------- | :-------- | | Total revenues | $2,096.0 | $1,846.3 | $1,860.5 | | Cost of sales | $483.7 | $470.5 | $524.3 | | Research and development (R&D) expense | $649.6 | $628.8 | $628.1 | | Selling, general and administrative (SG&A) expense | $854.0 | $759.4 | $737.7 | | Gain on sale of nonfinancial assets, net | $(108.0) | — | $(52.8) | | Provision for (benefit from) income taxes | $8.0 | $(11.3) | $(903.0) | | Net income (loss) | $141.6 | $(64.1) | $854.0 | [Business Developments](index=60&type=section&id=Business%20Developments) **BioMarin** advanced its commercial business and product pipeline in 2022, focusing on innovative rare disease therapies - **BioMarin** continued to grow its commercial business and advance its product candidate pipeline in 2022, aiming to develop and commercialize innovative therapies for rare diseases through internal research, acquisitions, and partnerships[775](index=775&type=chunk)[961](index=961&type=chunk) [Reorganization Plan](index=61&type=section&id=Reorganization%20Plan) **BioMarin** announced a reorganization in October 2022, reducing approximately **120 employees** and incurring **$23.0 million** in severance costs - On October 6, 2022, **BioMarin** announced a reorganization plan to simplify its organizational design, focusing resources on R&D pipeline advancement, maximizing commercial launch success, preparing for **ROCTAVIAN**'s EU and potential U.S. launch, and optimizing core infrastructure[565](index=565&type=chunk)[617](index=617&type=chunk)[964](index=964&type=chunk) - The reorganization resulted in a planned reduction of approximately **120 employees** (**4% of its global workforce**), mostly from U.S. operations, incurring **$23.0 million** in pre-tax severance and employee termination benefit charges in 2022[565](index=565&type=chunk)[617](index=617&type=chunk)[964](index=964&type=chunk) [2023 Outlook](index=61&type=section&id=2023%20Outlook) **BioMarin** expects to achieve 2023 operating objectives through pipeline investment and commercialization, anticipating net product revenue growth - **BioMarin** expects to achieve its 2023 operating objectives through product pipeline investments and commercial infrastructure support, including ongoing clinical studies for commercial products and early-stage candidates, and global commercialization of **VOXZOGO** and **ROCTAVIAN** in the EU[965](index=965&type=chunk) - The company anticipates growth in Net Product Revenues, excluding **KUVAN** due to market exclusivity loss, but continues to monitor macroeconomic conditions, reimbursement changes, market competition, and generic competitors[566](index=566&type=chunk)[966](index=966&type=chunk) [Uncertainty Relating to Macroeconomic Environment](index=60&type=section&id=Uncertainty%20Relating%20to%20Macroeconomic%20Environment) Macroeconomic conditions like inflation, interest rates, and supply chain disruptions could impact **BioMarin**'s operations and revenues - Macroeconomic conditions, including inflation, changes in interest and foreign currency exchange rates, natural disasters, ongoing COVID-19 effects, and supply chain disruptions, could impact **BioMarin**'s global revenue sources and business operations[561](index=561&type=chunk)[941](index=941&type=chunk) - The company is actively monitoring and managing its response to these conditions, assessing potential impacts on operating results and financial condition, and adjusting business processes to mitigate risks[941](index=941&type=chunk) [Critical Accounting Estimates](index=62&type=section&id=Critical%20Accounting%20Estimates) **BioMarin**'s financial statements rely on significant accounting estimates and judgments, regularly evaluated with the Audit Committee - **BioMarin**'s Consolidated Financial Statements rely on significant assumptions, judgments, and estimates that can impact net income/loss and reported amounts of assets, liabilities, revenue, and expenses[947](index=947&type=chunk) - Critical accounting policies and estimates are regularly evaluated and discussed with the Audit Committee, with historical assumptions generally not differing materially from actual results[947](index=947&type=chunk) [Results of Operations](index=64&type=section&id=Results%20of%20Operations) This section provides a detailed analysis of **BioMarin**'s total revenues for 2022, 2021, and 2020, including year-over-year changes Total Revenues (Millions USD) | Metric | 2022 | 2021 | 2020 | 2022 vs. 2021 Change | 2021 vs. 2020 Change | | :------------- | :-------- | :-------- | :-------- | :------------------- | :------------------- | | Total revenues | $2,096.0 | $1,846.3 | $1,860.5 | $249.7 | $(14.2) | [Financial Condition, Liquidity and Capital Resources](index=69&type=section&id=Financial%20Condition%2C%20Liquidity%20and%20Capital%20Resources) **BioMarin** expects sufficient liquidity for 12 months from cash and sales, managing **$1.1 billion** in **convertible debt** - **BioMarin** believes its cash generated from product sales, along with existing cash, cash equivalents, and investments, will be sufficient to meet liquidity requirements for at least the next 12 months[649](index=649&type=chunk) Cash, Cash Equivalents and Investments (Millions USD) | Category | Dec 31, 2022 | Dec 31, 2021 | Change | | :----------------------------------- | :----------- | :----------- | :------- | | Cash and cash equivalents | $724.5 | $587.3 | $137.2 | | Short-term investments | $567.0 | $426.6 | $140.4 | | Long-term investments | $333.9 | $507.8 | $(173.9) | | Total cash, cash equivalents and investments | $1,625.4 | $1,521.7 | $103.7 | Net Cash Flows (Millions USD) | Activity | 2022 | 2021 | 2022 vs. 2021 Change | | :-------------------------------- | :-------- | :-------- | :------------------- | | Net cash provided by operating activities | $175.9 | $304.5 | $(128.6) | | Net cash used in investing activities | $(20.0) | $(366.3) | $346.3 | | Net cash used in financing activities | $(18.7) | — | $(18.7) | - The company has **$1.1 billion** (undiscounted) in total **convertible debt** as of December 31, 2022, which impacts liquidity through semi-annual cash interest payments and principal repayment if not converted[1016](index=1016&type=chunk) [Item 7A. Quantitative and Qualitative Disclosure About Market Risk](index=71&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosure%20About%20Market%20Risk) **BioMarin** is exposed to market risks from foreign currency exchange rates, interest rates, and credit risks, mitigated by hedging and investment strategies - **BioMarin** is exposed to market risks from changes in foreign currency exchange rates, interest rates, and credit risks, which are mitigated through foreign currency derivative hedging, investment guidelines, and trade receivable monitoring[1020](index=1020&type=chunk)[1045](index=1045&type=chunk) - Approximately **51% of net product sales** and **19% of operating expenses** (excluding Cost of Sales) in 2022 were denominated in foreign currencies, primarily the Euro, making the company sensitive to USD fluctuations[626](index=626&type=chunk)[1046](index=1046&type=chunk) - A hypothetical **10% adverse movement** in foreign currency exchange rates could reduce the value of outstanding forward contracts by approximately **$83.2 million**[657](index=657&type=chunk) - The company's investment portfolio primarily consists of high-credit quality debt securities, and its **convertible debt** (2024 and 2027 Notes) has fixed interest rates, limiting exposure to rising interest rates[1022](index=1022&type=chunk)[1048](index=1048&type=chunk) [Item 8. Financial Statements and Supplementary Data](index=72&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section refers to the location of the audited consolidated financial statements and supplementary data within the Annual Report on Form 10-K - The financial statements and supplementary data are included in Part IV, Item 15 of this Annual Report on Form 10-K[1025](index=1025&type=chunk) [Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure](index=72&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20with%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) There are no changes in or disagreements with accountants on accounting and financial disclosure to report - There are no changes in or disagreements with accountants on accounting and financial disclosure[1027](index=1027&type=chunk) [Item 9A. Controls and Procedures](index=72&type=section&id=Item%209A.%20Controls%20and%20Procedures) **BioMarin**'s management concluded its disclosure controls and internal control over financial reporting were effective as of December 31, 2022, based on the **COSO 2013 Framework** - **BioMarin**'s management, including the CEO and CFO, concluded that its disclosure controls and procedures were effective as of December 31, 2022[1026](index=1026&type=chunk) - The company's internal control over financial reporting was assessed as effective at the reasonable assurance level as of December 31, 2022, based on the **COSO 2013 Framework**[631](index=631&type=chunk)[1052](index=1052&type=chunk) - No material changes in internal control over financial reporting occurred during the most recently completed quarter[663](index=663&type=chunk)[1028](index=1028&type=chunk) [Item 9B. Other Information](index=73&type=section&id=Item%209B.%20Other%20Information) No other information is required to be disclosed in this item - No other information is required to be disclosed in this item[1030](index=1030&type=chunk) [Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections](index=73&type=section&id=Item%209C.%20Disclosure%20Regarding%20Foreign%20Jurisdictions%20that%20Prevent%20Inspections) There are no disclosures regarding foreign jurisdictions that prevent inspections - Not applicable[1030](index=1030&type=chunk) Part III [Item 10. Directors, Executive Officers and Corporate Governance](index=74&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information regarding directors, executive officers, and corporate governance is incorporated by reference from the company's 2023 annual meeting of stockholders proxy statement - Information on directors, executive officers, and corporate governance is incorporated by reference from the proxy statement for the 2023 annual meeting of stockholders[665](index=665&type=chunk)[1031](index=1031&type=chunk) [Item 11. Executive Compensation](index=74&type=section&id=Item%2011.%20Executive%20Compensation) Information regarding executive compensation is incorporated by reference from the company's 2023 annual meeting of stockholders proxy statement - Information on executive compensation is incorporated by reference from the proxy statement for the 2023 annual meeting of stockholders[666](index=666&type=chunk)[1091](index=1091&type=chunk) [Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=74&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Information regarding security ownership of certain beneficial owners, management, and related stockholder matters is incorporated by reference from the company's 2023 annual meeting of stockholders proxy statement - Information on security ownership of certain beneficial owners, management, and related stockholder matters is incorporated by reference from the proxy statement for the 2023 annual meeting of stockholders[636](index=636&type=chunk)[1032](index=1032&type=chunk) [Item 13. Certain Relationships and Related Transactions and Director Independence](index=74&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%20and%20Director%20Independence) Information regarding certain relationships, related transactions, and director independence is incorporated by reference from the company's 2023 annual meeting of stockholders proxy statement - Information on certain relationships, related transactions, and director independence is incorporated by reference from the proxy statement for the 2023 annual meeting of stockholders[637](index=637&type=chunk)[1058](index=1058&type=chunk) [Item 14. Principal Accounting Fees and Services](index=74&type=section&id=Item%2014.%20Principal%20Accounting%20Fees%20and%20Services) Information regarding principal accounting fees and services is incorporated by reference from the company's 2023 annual meeting of stockholders proxy statement - Information on principal accounting fees and services is incorporated by reference from the proxy statement for the 2023 annual meeting of stockholders[667](index=667&type=chunk)[1033](index=1033&type=chunk) Part IV [Item 15. Exhibits, Financial Statement Schedules](index=75&type=section&id=Item%2015.%20Exhibits%2C%20Financial%20Statement%20Schedules) This section lists all exhibits and financial statement schedules filed as part of the Annual Report on Form 10-K, including the Exhibit Index, Consolidated Financial Statements, and XBRL documents - This item includes the Exhibit Index, Financial Statements, and XBRL Instance Document, Taxonomy Extension Schema, Calculation, Definition, Labels, and Presentation Link Documents[638](index=638&type=chunk)[641](index=641&type=chunk)[1034](index=1034&type=chunk)[1037](index=1037&type=chunk) [Item 16. Form 10-K Summary](index=79&type=section&id=Item%2016.%20Form%2010-K%20Summary) This item indicates that the Form 10-K Summary is not applicable or not provided as a separate section - This item is typically a summary of the Form 10-K, but the content provided does not contain a summary[671](index=671&type=chunk)[1063](index=1063&type=chunk)

Table of Contents Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, par value $0.001 BMRN The Nasdaq Global Select Market ______________________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ______________________________________ Form 10-Q ______________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANG ...

Biomarin Pharmaceutical, Inc. (NASDAQ:BMRN) Q3 2022 Earnings Conference Call October 26, 2022 4:30 PM ET Company Participants Traci McCarty - VP, IR J.J. Bienaime - Chairman & CEO Jeffrey Ajer - EVP & Chief Commercial Officer Henry Fuchs - President, Worldwide Research & Development Brian Mueller - EVP, Finance & CFO Conference Call Participants Philip Nadeau - Cowen and Company Salveen Richter - Goldman Sachs Jessica Fye - JPMorgan Chase & Co. Srikripa Devarakonda - Truist Securities Paul Matteis - Stifel, ...

Table of Contents Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, par value $0.001 BMRN The Nasdaq Global Select Market ______________________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ______________________________________ Form 10-Q ______________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANG ...

Call Start: 16:30 January 1, 0000 5:42 PM ET BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Q2 2022 Earnings Conference Call August 3, 2022 16:30 ET Company Participants Traci McCarty - Vice President of Investor Relations J.J. Bienaime - Chairman and Chief Executive Officer Jeff Ajer - Executive Vice President and Chief Commercial Officer Hank Fuchs - President, Worldwide Research and Development Brian Mueller - Executive Vice President and Chief Financial Officer Greg Guyer - Executive Vice President and Chie ...

Table of Contents Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, par value $0.001 BMRN The Nasdaq Global Select Market ______________________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ______________________________________ Form 10-Q ______________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANG ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Q1 2022 Earnings Conference Call April 27, 2022 4:30 PM ET Company Participants Traci McCarty - Vice President, Investor Relations J.J. Bienaime - Chairman and Chief Executive Officer Jeff Ajer - Executive Vice President and Chief Commercial Officer Hank Fuchs - President, Worldwide Research and Development Greg Guyer - Executive Vice President and Chief Technical Officer Brian Mueller - Executive Vice President and Chief Financial Officer Conference Call Participa ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ____________ Form 10-K ____________ (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 Or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to . Commission file number: 000-26727 ____________ BioMarin Pharmaceutical Inc. (Exact name of registrant as specified in its charter) D ...