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BioMarin Announces Oral Presentation of 2-Year Analysis of Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A at 15th Annual Congress of European Association for Haemophilia and Allied Disorders (EAHAD) 2-4 February

Published: 2022-02-04 16:15:00 ET

- Improved Bleed Free Rate from 32% at Baseline Maintained Through Year 1 (82%) and Year 2 (84%)

- Factor VIII Treatment Burden Compared to Baseline Reduced by 98%

- 95% of Study Participants Remain Off Standard of Care Factor VIII Prophylactic Therapy

SAN RAFAEL, Calif., Feb. 4, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Company presented positive results from a two-year analysis of the Phase 3 GENEr8-1 study and an overall safety update of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). 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.