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 hold a conference call on February 27 at 4:30 p.m. ET to discuss its fourth quarter and full-year 2022 financial results. CEO Jean-Jacques Bienaimé will lead the call, providing a general business update. Investors can dial in using the U.S. number 800-831-4163 or international number 213-992-4616. A replay will be available after the call. BioMarin, founded in 1997, focuses on developing targeted therapies for rare genetic disorders, with a strong pipeline of commercial and clinical candidates addressing significant unmet medical needs.
BioMarin Pharmaceutical announced promising results from the Phase 3 GENEr8-1 study of ROCTAVIAN, a one-time gene therapy for severe hemophilia A, with data covering over three years. Key findings include an 80% reduction in annualized bleed rates and a 94% decrease in Factor VIII usage. Notably, 92% of patients remained off prophylactic treatment by Year 3. The company signed its first outcomes-based agreement in Germany, with more expected soon. Additionally, the FDA completed a pre-license inspection of BioMarin's manufacturing facility, further supporting ROCTAVIAN's regulatory review.
BioMarin Pharmaceutical (NASDAQ: BMRN) will present at the 41st Annual J.P. Morgan Healthcare Conference on January 9, 2023, at 10:30 am PT in San Francisco, California. CEO Jean-Jacques Bienaimé will lead the presentation. Interested investors can access the live webcast on www.biomarin.com. The replay will be available for a week post-event.
BioMarin has submitted a Supplemental New Drug Application (sNDA) to the FDA to expand the use of VOXZOGO for treating children with achondroplasia under age 5. The submission follows positive results from a Phase 2 study showing increased growth velocity across all age cohorts. The company aims to help over 1,000 eligible children in the U.S. and Europe. Regulatory decisions are anticipated in the second half of 2023. VOXZOGO is already approved for older children with the same condition and aims to support growth in those with open growth plates.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced significant progress in the FDA review of its Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec), a gene therapy for severe hemophilia A. The FDA has decided not to hold an advisory committee meeting as previously planned, which could expedite the approval process. Additionally, BioMarin is preparing for upcoming FDA manufacturing inspections. The therapy has received RMAT designation, and clinical studies are ongoing, indicating potential for a transformative treatment option for patients with this condition.
BioMarin Pharmaceutical (BMRN) announced a progress update on its Biologics License Application for valoctocogene roxaparvovec gene therapy for severe Hemophilia A. The FDA has scheduled a Pre-Licensure Inspection at its Novato, CA facility. Additionally, the FDA requested a three-year data analysis from the Phase 3 GENEr8-1 study, which could potentially extend the current PDUFA target action date of March 31, 2023, by three months if deemed a major amendment. BioMarin expressed optimism regarding the initial bleed control results from patients in the study.
BioMarin Pharmaceutical (BMRN) announced participation in two major investor conferences in November 2022. The Credit Suisse 31st Annual Healthcare Conference is scheduled for November 8 at 8:35 am PT in Rancho Palos Verdes, CA. The 13th Annual Jefferies Global Healthcare Conference will take place on November 16 at 8:35 am GMT in London, England. Live webcasts will be available on the company's investor website, with archived presentations accessible afterward.
BioMarin reported third-quarter 2022 total revenues of $505.3 million, a 24% increase year-over-year, driven by strong sales of VOXZOGO, which generated $48 million in the quarter. The company raised its full-year VOXZOGO revenue guidance to between $140 million and $170 million. Meanwhile, the FDA accepted the BLA for ROCTAVIAN, with a target action date of March 31, 2023. Despite a GAAP net loss of $6.7 million, non-GAAP income rose to $82.7 million, highlighting the company's ongoing financial resilience.
BioMarin Pharmaceutical announced the FDA's acceptance of its Biologics License Application (BLA) for the gene therapy valoctocogene roxaparvovec for adults with severe hemophilia A. This therapy, if approved by the PDUFA target date of March 31, 2023, would be the first of its kind in the U.S. BioMarin's application includes extensive clinical data demonstrating effective bleed control. The FDA previously granted Regenerative Medicine Advanced Therapy and Breakthrough Therapy designations for this treatment, highlighting its potential to address unmet medical needs.
BioMarin Pharmaceutical (Nasdaq:BMRN) announced an organizational redesign aimed at enhancing operational efficiency and focusing on R&D investments. This restructuring will lead to a reduction of approximately 120 employees, about 4% of its workforce, resulting in annual cost savings of $50 million from 2023. A one-time cost of $20-$25 million will be incurred due to severance. The company plans to reinvest savings into its product pipeline, including the potential U.S. launch of ROCTAVIAN™ and the continued success of VOXZOGO®.