Core Insights - The FDA has approved BioMarin Pharmaceutical's supplemental Biologics License Application for PALYNZIQ to include pediatric patients aged 12 and older with phenylketonuria (PKU), making it the only enzyme substitution therapy for this condition [1][5][29] Group 1: FDA Approval and Study Results - The approval is based on the Phase 3 PEGASUS study, which demonstrated that PALYNZIQ significantly reduced blood phenylalanine (Phe) levels compared to diet alone in adolescents aged 12 to less than 18 with uncontrolled blood Phe concentrations greater than 600 µmol/L [2][3] - Participants in the PALYNZIQ group showed a mean reduction in blood Phe levels from 1025 µmol/L at baseline to 567 µmol/L at Week 72, while the diet-only group had a reduction from 1029 µmol/L to 973 µmol/L [3] - By the end of Part 1 of the study, 44.4% of participants reached blood Phe levels below guideline recommendations, with an average reduction of 828 µmol/L, representing a 94% reduction from baseline [3] Group 2: Clinical Implications and Patient Management - PALYNZIQ allows for an unrestricted diet while effectively managing blood Phe levels, which is particularly beneficial for adolescents facing increased independence and academic demands [2][5] - The treatment has shown higher adherence rates in adolescents living at home with family support, making it a promising option for this demographic [2] - The most common adverse reactions in adolescents included injection site reactions, arthralgia, headache, and hypersensitivity reactions, with a notable safety profile observed during the maintenance phase [4][22] Group 3: Company Background and Future Directions - BioMarin has been actively working with medical and advocacy communities for over two decades to improve the lives of individuals with PKU, and the expansion of PALYNZIQ's approval is a continuation of this effort [5][28] - The company is also pursuing approval from the European Medicines Agency to extend PALYNZIQ treatment to adolescents aged 12 and older in the European Union [5][29] - BioMarin is recognized as a leading biotechnology company focused on rare diseases, with a strong pipeline of therapies aimed at genetically defined conditions [28]

侏儒症的治疗，正迎来被全面颠覆的时刻。 作为最常见的侏儒症类型，软骨发育不全（ACH）过去很长时间无特效疗法，直到2021年，"孤儿药之王"BioMarin研 发的Vosoritide，作为首个获批药物，才开启了药物对症治疗时代。凭借独家优势，其2025年销售额高达9.27亿美元。 但这种垄断格局，即将被打破，多款在研新药正从作用机制到给药方式，向Vosoritide发起挑战。Ascendis的TransCon- CNP通过包裹技术延长了药物作用时间，实现了周剂给药，目前正处于FDA审批阶段；而BridgeBio的口服药 Infigratinib，则从作用机制到用药体验实现全面突破。 Infigratinib靶向的FGFR3（成纤维细胞生长因子受体3）正是驱动ACH疾病发生的关键靶点，2月12日，BridgeBio公布 Infigratinib在ACH中取得的首个具有统计学显著改善意义的3期顶线结果，公司计划下半年向FDA提交新药申请。 获批后的长期扩展临床数据显示，Vosoritide的生长促进效应可持续至少7年。 尽管目前全球仅有5000名婴幼儿使用该药，但罕见病药物的高定价模式，叠加长期持续给药的治疗需 ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is among the 20 Best Investments in 2026. BioMarin Pharmaceutical Inc. (BMRN) Seen as Attractive Buy Ahead of Transcon-CNP PDUFA, Says Wells Fargo BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is next on our list. TheFly reported on February 18 that Wells Fargo & Company increased its price target for BMRN from $70 to $75 while keeping an Overweight rating. The firm believes the upcoming Transcon-CNP PDUFA may mark the final negative catalyst for BMRN, and its sum ...

Financial Commitments and Acquisitions - The company entered into a debt financing commitment for approximately $3.7 billion to fund the acquisition of Amicus, which was later reduced to $2.8 billion after issuing $850 million in senior unsecured notes[25]. - The company plans to enter into a senior secured term loan facility for approximately $2.8 billion and a new $600 million revolving credit facility in 2026[25]. - The company entered into a Bridge Commitment for up to approximately $3.7 billion in debt financing related to the pending acquisition of Amicus[436]. - In February 2026, the company issued $850.0 million in aggregate principal amount of the 2034 Notes to finance the pending acquisition of Amicus, reducing the Bridge Commitment from approximately $3.7 billion to $2.8 billion[437]. Revenue and Product Performance - In 2025, net product revenues for VOXZOGO reached $926.9 million, while VIMIZIM generated $792.1 million, and NAGLAZYME brought in $485.4 million[28]. - Total revenues for 2025 reached $3,221.3 million, an increase of 12.7% compared to $2,853.9 million in 2024[412]. - The company recognized net product revenues from ALDURAZYME sales, receiving payments ranging from 39.5% to 50% based on sales volume[446]. - The gross-to-net sales adjustments for the year ended December 31, 2025, included a balance at the beginning of the year of $195.0 million and an ending balance of $246.1 million[448]. Research and Development - Total R&D expense for 2025 was $921.9 million, up 23.4% from $747.2 million in 2024, driven by higher spending on later-stage clinical programs[416]. - R&D expense for later-stage clinical programs surged to $308.3 million in 2025, a significant increase from $27.6 million in 2024[416]. - The company incurred a $221.0 million In-Process Research and Development (IPR&D) charge following the Inozyme acquisition in 2025[416]. - The company reported a decrease in R&D spending on marketed products, primarily related to ROCTAVIAN, which fell to $229.9 million in 2025 from $285.6 million in 2024[416]. Market and Competitive Landscape - The biopharmaceutical industry is highly competitive, with potential competition from both larger and smaller companies in various therapeutic areas[65]. - There are currently no approved drugs for ENPP1 deficiency in the U.S. or EU, indicating a potential market opportunity for BMN 401[79]. - The commercial organization is structured around two business units: Skeletal Conditions (VOXZOGO) and Enzyme Therapies, with ongoing assessments for additional market agreements[60]. Regulatory and Compliance - The approval process for new drugs typically takes many years, involving preclinical tests and multiple phases of clinical trials[89][96]. - Clinical trials must comply with regulations and good clinical practices, with oversight from regulatory agencies and ethics committees[93][94]. - The FDA's Fast Track program allows for expedited review of drugs addressing unmet medical needs, with a determination made within 60 days of request[100]. - The FDA may approve drugs for serious conditions based on surrogate endpoints that predict clinical benefits, subject to post-marketing compliance requirements[102]. Financial Performance and Expenses - Cost of sales increased to $717.4 million in 2025, primarily due to a $119.2 million write-off of ROCTAVIAN inventory[412]. - Gross margin decreased to 77.7% in 2025 from 79.7% in 2024, largely impacted by the ROCTAVIAN inventory write-off[412]. - Total SG&A expenses for 2025 were $1,153.0 million, an increase of 14.3% from $1,009.0 million in 2024[418]. - G&A expenses rose to $622.8 million in 2025, a 17.0% increase from $532.3 million in 2024, primarily due to $118.5 million in restructuring charges related to ROCTAVIAN[419]. Legal and Intellectual Property - The company is engaged in legal actions to protect its intellectual property, particularly concerning patents related to VOXZOGO[81]. - The patent term for U.S. drugs can be extended up to five years under the Hatch-Waxman Act, providing additional market exclusivity[82]. - ALDURAZYME and NAGLAZYME patents expired in November 2020 and November 2023, respectively, with ongoing efforts to pursue additional patents and extensions[83]. Employee and Workforce - Employee count as of December 31, 2025, was 3,221, with 2,026 employees located in the U.S. and Canada[149]. - The company is committed to leveraging diverse perspectives to drive innovation and growth within its workforce[152]. Tax and Financial Obligations - Provision for income taxes increased to $133.6 million in 2025, up from $114.9 million in 2024, mainly due to non-deductible acquired IPR&D expenses[426]. - The liability for unrecognized tax benefits was $380.9 million as of December 31, 2025, with uncertain timing of future payments[443].

Key Takeaways BioMarin reported Q4 EPS of 46 cents, beating estimates while revenues rose 17% Y/Y to $875M.Voxzogo sales jumped 31% to $273M in Q4, while BioMarin's enzyme therapies sales rose 13%.BioMarin will withdraw Roctavian after failing to find a buyer, recording a $119.2M charge in Q4.BioMarin Pharmaceutical (BMRN) reported fourth-quarter 2025 adjusted earnings per share of 46 cents, beating the Zacks Consensus Estimate of 25 cents. However, earnings declined 50% year over year, largely due to a $11 ...

BioMarin Pharmaceutical (NasdaqGS:BMRN) Q4 2025 Earnings call February 23, 2026 04:30 PM ET Company ParticipantsAlexander Hardy - CEOBrian Mueller - CFOCristin Hubbard - Chief Commercial OfficerGena Wang - Managing Director of Biotech Equity ResearchGreg Friberg - Chief Research and Development OfficerJason Gerberry - Managing DirectorMohit Bansal - Managing Director and Co-Head of Therapeutics ResearchPaul Matteis - Managing DirectorTraci McCarty - Head of Investor RelationsConference Call ParticipantsChri ...

BioMarin Pharmaceutical (NasdaqGS:BMRN) Q4 2025 Earnings call February 23, 2026 04:30 PM ET Speaker10Thank you for standing by. My name is Kate, and I will be your conference operator today. At this time, I would like to welcome everyone to the BioMarin Pharmaceutical fourth quarter and full year 2025 conference call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this ti ...

Fourth Quarter and Full Year 2025 Earnings Q4'25 and FY'25 Earnings Agenda: Alexander Hardy Chief Executive Officer Brian Mueller Chief Financial Officer Cristin Hubbard Chief Commercial Officer February 23, 2026 Forward-Looking Statements This presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expect ...

In its upcoming report, BioMarin Pharmaceutical (BMRN) is predicted by Wall Street analysts to post quarterly earnings of $0.25 per share, reflecting a decline of 72.8% compared to the same period last year. Revenues are forecasted to be $829.66 million, representing a year-over-year increase of 11%.Over the last 30 days, there has been a downward revision of 2.3% in the consensus EPS estimate for the quarter, leading to its current level. This signifies the covering analysts' collective reconsideration of ...

BioMarin to Host Fourth Quarter and Full-Year 2025 Financial Results Conference Call and Webcast on Monday, February 23, 2026, at 4:30pm ET [Accessibility Statement] Skip NavigationSAN RAFAEL, Calif., Feb. 17, 2026 /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 Monday, February 23, 2026, at 4:30 p.m. ET to discuss fourth quarter and full-year 2025 financial results ...