Welcome to our dedicated page for Biomarin Pharmaceutical news (Ticker: BMRN), a resource for investors and traders seeking the latest updates and insights on Biomarin Pharmaceutical stock.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is a global rare disease biotechnology company focused on medicines for genetically defined conditions. This news page aggregates company announcements, press releases and event updates so readers can follow how BioMarin’s strategy, pipeline and commercial portfolio evolve over time.
Recent communications from BioMarin highlight its emphasis on Enzyme Therapies and Skeletal Conditions business units, as well as its work with ROCTAVIAN, a gene therapy for hemophilia A. The company issues news on topics such as financial results, long-term guidance, corporate strategy, business development transactions, clinical data presentations and regulatory milestones for its therapies.
Examples of news themes include updates on VOXZOGO in achondroplasia and other skeletal conditions, progress with PALYNZIQ in phenylketonuria, development of BMN 401 for ENPP1 deficiency and other pipeline programs in rare genetic diseases. BioMarin also reports on strategic partnerships, such as digital and data collaborations, and on planned acquisitions intended to expand and diversify its rare disease portfolio.
Investors and followers of BMRN can use this page to review earnings announcements, conference presentations, investor day materials and product-specific updates released through newswires. By checking this feed regularly, readers can see how BioMarin describes its growth priorities, clinical milestones and capital allocation decisions across its rare disease franchises.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host a conference call and webcast on October 26, 2022, at 4:30 p.m. ET to discuss its third quarter 2022 financial results and provide a general business update. This follows the market's closure on the same day. Interested participants can access the event via BioMarin's investor section on their website. The company focuses on developing innovative therapies for serious genetic diseases, with eight commercial products and numerous candidates in development.
BioMarin Pharmaceutical has resubmitted its Biologics License Application (BLA) for valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, to the FDA. This resubmission addresses the FDA's previous Complete Response Letter and includes extensive clinical data, including the global GENEr8-1 Phase 3 study outcomes. If approved, it would become the first gene therapy for this condition in the U.S. The FDA is expected to respond by late October, and the review process could extend beyond the typical six months due to the volume of data provided.
BioMarin has announced that the Institute for Clinical and Economic Review (ICER) reports that its gene therapy Roctavian offers over $4 million in lifetime savings compared to emicizumab for hemophilia A treatment. The therapy, priced at $2.5 million, was evaluated in a model accounting for various treatment costs. BioMarin plans to resubmit its Biologics License Application (BLA) to the FDA by the end of September 2022, with anticipated European sales starting in Q4 2022. The findings from ICER are subject to public comment before a final report is issued.
BioMarin Pharmaceutical announced the European Commission's conditional marketing authorization for ROCTAVIAN™ (valoctocogene roxaparvovec), the first gene therapy for severe hemophilia A. This authorization offers 10 years of market exclusivity based on the therapy's potential significant benefits over existing treatments. ROCTAVIAN is expected to reduce the burden of regular infusions for patients. The approval is supported by extensive clinical data, including results from the GENEr8-1 Phase 3 study, which showed durable bleed control. BioMarin plans a BLA resubmission in the U.S. by the end of September 2022.
BioMarin reported record revenues of $533.8 million for Q2 2022, a 6% increase year-over-year, aided by strong growth from Voxzogo and other franchises. Total revenues for the first half surpassed $1 billion, prompting the company to raise its full-year guidance. Notably, Voxzogo generated $34.4 million in product revenues, while Kuvan revenues declined by 27%. BioMarin anticipates the approval of Roctavian in Europe by Q3 2022 and plans to resubmit its BLA in the U.S. for Roctavian by September.
BioMarin Pharmaceutical (NASDAQ: BMRN) will host a conference call and webcast on August 3, 2022, at 4:30 p.m. ET to discuss its second quarter 2022 financial results and provide a business update. The call can be accessed via U.S. and international dial-in numbers, with a replay available for one week following the event. BioMarin specializes in developing therapies for serious rare diseases, with a portfolio of seven commercial products and multiple candidates in various stages of development.
BioMarin Pharmaceutical presented findings on valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, at the ISTH 2022 World Congress in London. The data support its efficacy and safety over six years, with a potential European Commission approval expected in Q3 2022 following a positive CHMP recommendation. This gene therapy may significantly reduce the burden of disease and treatment for patients. The presentations included comparative effectiveness studies and hemostatic results, showcasing the company's commitment to advancing care for individuals with hemophilia A.
BioMarin Pharmaceutical has received a positive recommendation from the European Medicines Agency's CHMP for its gene therapy, valoctocogene roxaparvovec, targeting severe hemophilia A in adults. This therapy, branded as ROCTAVIAN™, is set to be the first gene therapy approved in Europe for this condition. With over 20,000 adults affected across Europe, the Middle East, and Africa, the company anticipates increased patient access post-approval. The European Commission's final decision is expected in Q3 2022, contingent on the ongoing collection of long-term clinical data.
BioMarin Pharmaceutical announced that Japan's Ministry of Health has approved VOXZOGO (vosoritide) for treating achondroplasia in children of all ages with open growth plates. This follows previous approvals in the U.S., Europe, and Brazil. VOXZOGO works by downregulating FGFR3 signaling to promote bone growth. The decision was based on successful Phase 3 and Phase 2 clinical trials. With around 1,500 potential patients in the Asia-Pacific region, Japan accounts for about half, making this approval significant in expanding treatment access.
BioMarin Pharmaceutical (NASDAQ:BMRN) presented promising results from a Phase 2 trial of VOXZOGO™ (vosoritide) for treating achondroplasia in children under five. Over 52 weeks, children on VOXZOGO (n=43) showed a height Z-score improvement of 0.30 SD and an annualized growth velocity increase of 0.92 cm/year compared to placebo (n=32). The company plans discussions with health authorities in H2 2022 to expand access for younger patients. The safety profile was consistent with older patients, with fewer serious adverse events in the Voxzogo group, suggesting early treatment benefits.