Biomarin Pharmaceutical Inc Stock Price, News & Analysis

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.

BioMarin Pharmaceutical announced significant results from its Phase 1/2 study of valoctocogene roxaparvovec, showing a 95% reduction in mean annualized bleed rate (ABR) and 96% reduction in Factor VIII usage after six years for the 6e13 vg/kg cohort. The 4e13 vg/kg cohort exhibited similar success with a 91% reduction in mean ABR over five years. BioMarin plans to present data at the ISTH 2022 Congress and expects to resubmit its Biologics Licensing Application (BLA) to the FDA by the end of September 2022, while the EMA's Marketing Authorization Application is currently under review.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has appointed Humaira Serajuddin as Senior Vice President, Chief Marketing Officer, and Erin Burkhart as Group Vice President, Chief Accounting Officer. These strategic hires are aimed at supporting the company's growth and navigating towards sustainable profitability. Serajuddin brings over 20 years of pharmaceutical experience, including successful product launches, while Burkhart has extensive knowledge in corporate finance and accounting, having previously worked at Eli Lilly. Both leaders are expected to drive BioMarin's anticipated growth and transformation in the coming years.

BioMarin Pharmaceutical announced the presentation of findings from their ongoing studies of valoctocogene roxaparvovec, an investigational gene therapy for severe hemophilia A, at the WFH 2022 World Congress in Montreal from May 8-11. The data includes updates on treatment durability, hepatotoxicity, and quality of life for patients. The global phase 3 GENEr8-1 study showed positive two-year results. BioMarin aims to improve shared decision-making for patients with severe hemophilia A, as current treatments often don't eliminate bleeding risks.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that CEO Jean-Jacques Bienaimé will present at the BofA Securities Healthcare Conference on May 12, 2022, at 8:40 am PT/11:40 am ET in Las Vegas. Interested individuals can access the live webcast on the BioMarin website. A replay will be available for 90 days post-event. BioMarin specializes in developing therapies for serious rare diseases, with a portfolio of seven commercial products and various clinical candidates.

BioMarin reported record total revenues of $519 million for Q1 2022, an 11% increase year-over-year, driven by strong demand for Voxzogo and other products. Notably, Voxzogo generated $20 million in net product revenues, with full-year guidance revised to $100-125 million. GAAP net income surged to $120.8 million, attributed to a $89 million gain from a Rare Pediatric Disease Voucher. The company's pipeline includes valoctocogene roxaparvovec for hemophilia A, with regulatory reviews ongoing in Europe and the U.S.