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BioMarin Announces Stable and Durable Annualized Bleed Control in the Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; 134-Participant Study Met All Primary and Secondary Efficacy Endpoints at Two Year Analysis

Published: 2022-01-09 20:13:00 ET

- Annualized Bleeding Rate (ABR) Reduced by 85% from Baseline, Demonstrating Superiority to Factor VIII Prophylaxis

- Mean Factor VIII Activity of 23 IU/dL (Chromogenic Assay); 36 IU/dL (One-Stage Assay) Observed at Year 2

- Company Plans to Present Additional Data at Upcoming Medical Meetings

- Marketing Authorization Application under review with the European Medicines Agency (EMA) and Regulatory Submission to FDA Expected in 2Q 2022

- Conference Call and Webinar to be Held Today, Sunday, January 9, 2022 at 5:00 PM Eastern Time

SAN RAFAEL, Calif., Jan. 9, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in hemophilia, with 134 participants.

In the GENEr8-1 Phase 3 study, Annualized Bleeding Rate (ABR) was significantly reduced by 4.1 treated bleeds per year (p-value

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All statements made via social media sites sponsored or maintained by Enhanced Investments and its affiliates are for informational purposes only and do not constitute a comprehensive description of Enhanced Investments' investment advisory services.
Certain investments are not suitable for all investors. Before investing, consider your investment objectives and applicable fees. The rate of return on investments can vary widely over time, especially for long term investments. Investment losses are possible, including the potential loss of all amounts invested. Information provided by Enhanced Investments is for informational and general educational purposes only and is not investment or financial advice.