Biomarin Pharmaceutical Inc Stock Price, News & Analysis

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will participate virtually in the SVB Securities Global Biopharma Conference on February 16, 2023, at 11:20 a.m. ET. Company leaders, Jean-Jacques Bienaimé and Brian Mueller, will represent the firm. Investors can access the live webcast at BioMarin's investor website. An archived version will also be available for a limited time post-conference. BioMarin, founded in 1997, focuses on genetic therapies and has developed eight commercial therapies for rare genetic disorders to meet significant medical needs. For more information, visit www.biomarin.com.

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.