Biomarin Pharmaceutical Inc Stock Price, News & Analysis

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®.

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.