Financial Performance and Guidance - Total revenue for 2024 is estimated to be $555 million to $560 million, exceeding the updated guidance range and representing approximately 29% growth versus 2023 [7] - Crysvita revenue for 2024 is estimated to be $405 million to $410 million, representing approximately 24% growth versus 2023 [7] - Dojolvi revenue for 2024 is estimated to be $87 million to $89 million, representing approximately 25% growth versus 2023 [7] - 2025 total revenue is expected to be between $640 million and $670 million, with a growth of approximately 14-20% compared to 2024 [1][6] - Cash, cash equivalents, and available-for-sale investments were approximately $745 million as of December 31, 2024 [1] Clinical Trials and Pipeline Updates - Phase 3 Orbit and Cosmic clinical trials for setrusumab in pediatric and young adult patients with osteogenesis imperfecta (OI) are ongoing, with the second interim analysis expected in mid-2025 and a potential final analysis in Q4 2025 [2][8] - UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) has a Biologics License Application (BLA) submitted, with a PDUFA decision and launch expected in the second half of 2025 [3] - UX701 AAV gene therapy for Wilson Disease is in Phase 1/2/3 study, with Cohort 4 enrollment expected to complete in the second half of 2025 [5][15] - GTX-102 Phase 3 Aspire study for Angelman syndrome is enrolling, with completion expected in the second half of 2025 [13] - DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa) is expected to file a BLA in mid-2025 [14] - DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency is in Phase 3 study, with enrollment completion expected in early 2025 [16] Strategic Outlook and Milestones - The company anticipates important pivotal Phase 3 results in osteogenesis imperfecta and completion of enrollment in the Phase 3 trial for Angelman syndrome in 2025 [11] - The company is preparing for the potential launch of its first gene therapy for Sanfilippo syndrome and filing a BLA for its second gene therapy for Glycogen Storage Disease Type Ia in 2025 [11] - The company expects to potentially launch three to four new therapies over the next couple of years, accumulating a total of eight to nine approved products over a 10-year period [11] Corporate Overview - Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases [17] - The company has a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need [17] - The company is led by a management team experienced in the development and commercialization of rare disease therapeutics [18]

Ultragenyx Pharmaceutical (RARE) announced that the European Commission (EC) has expanded the eligible patient population for one of its marketed products, Evkeeza (evinacumab), an ANGPTL3 inhibitor. Per the latest approval, Evkeeza is now indicated as an adjunct to diet and other lipid-lowering therapies to treat children aged six months and older with homozygous familial hypercholesterolemia (HoFH). The EC’s approval makes Evkeeza the first HoFH medicine indicated for this pediatric population in the EU.H ...

NOVATO, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 AM PT. The live and archived webcast of the presentation will b ...

Core News Summary - The European Commission (EC) has extended the approval of Evkeeza (evinacumab) for treating children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH), making it the first and only medicine approved in the EU for this age group [1][2] - Evkeeza is an ANGPTL3 inhibitor that reduces LDL-C levels and is now indicated for pediatric patients as young as 6-months old, addressing a critical unmet need in managing HoFH [1][5] Efficacy and Safety - Model-based extrapolation analysis predicts that pediatric patients aged 6-months to less than 5 years will experience a similar or higher magnitude of LDL-C reduction at week 24 compared to adults when receiving a 15 mg/kg dose every 4 weeks [2] - Compassionate use data for five patients aged 1-4 years showed clinically meaningful LDL-C reductions consistent with older patients, with no new safety concerns identified [2] Market and Availability - Evkeeza is now reimbursed and commercially available in the UK, US, Canada, Italy, Japan, the Netherlands, Spain, and Luxembourg, with early access schemes in 13 additional countries including Austria and France [2] - The treatment was initially approved for adults and adolescents aged 12+ in June 2021, with the indication extended to children aged 5-11 in December 2023 [2] Disease Background - HoFH is an ultrarare genetic disorder affecting 1 in 300,000 people globally and approximately 1,600 in the EU, characterized by dangerously high LDL-C levels (>400 mg/dL) and early cardiovascular risks [3][4] Product Mechanism - Evkeeza works by blocking the ANGPTL3 protein, which regulates cholesterol levels, and is administered via monthly infusions [5] Development and Commercialization - Evkeeza was discovered and developed by Regeneron Pharmaceuticals, Inc, with Ultragenyx responsible for its development and commercialization outside the US [7] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing novel therapies for rare and ultrarare genetic diseases, with a diverse portfolio addressing high unmet medical needs [12][13]

Ultragenyx Pharmaceutical (RARE) announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway.Earlier this year, the FDA aligned with Ultragenyx, after a thorough evaluation, on the use of cerebral spinal fluid (CSF) heparan sulfate (HS) as a well-characterized biomarker to support an accelerated approval pathway for mucopolysaccharidoses ...

LONDON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that NHS England has implemented the commissioning of Evkeeza (evinacumab) following the National Institute for Health and Care Excellence (NICE) final guidance in September. The use of Evkeeza in eligible people aged 12 years and older will be routinely commissioned by ...

Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study (NCT06617429) evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome. "Initiation of patient dosing in our Phase 3 Aspire stu ...

If approved, UX111 would be the first approved therapy in the U.S. for Sanfilippo Syndrome Type ANOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a B ...

PLYMOUTH MEETING, Pa., Dec. 3, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced that Harmony's management team will participate in a fireside chat at the upcoming Oppenheimer Movers in Rare Disease Summit in New York, NY on Thursday, December 12, 2024, at 9:00 a.m. ET.A webcast of the fireside chat will be available on the investor page of Harmony's website at https://ir.harmonybiosciences.com/.About Harmony BiosciencesHarmony Biosciences is a pharmaceutical company de ...

NOVATO, Calif., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences. Citi’s 2024 Global Healthcare Conference (Miami, FL) Tuesday, December 3, 2024, Emil Kakkis, M.D., Ph.D., CEO and President and Howard Horn, Chief Financial Officer, will participate in a fireside cha ...