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BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A on聽August 18, 2020. 聽The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.

Having previously agreed with the Agency on the extent of data necessary to support the BLA, the FDA introduced a new recommendation for two years of data from the Company’s ongoing 270-301 study (Phase 3) to provide聽substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint.聽 The Agency first informed the Company of this recommendation in the CRL having not raised this at any time during development or review.聽 The Agency recommended that the Company complete the Phase 3 Study and submit two-year follow-up safety and efficacy data on all study participants.聽 FDA concluded that the differences between Study 270-201 (Phase 1/2) and the Phase 3 study limited its ability to rely on the Phase 1/2 study to support durability of effect.聽 The Phase 3 study was fully enrolled in聽November 2019, and the last patient will complete two years of follow up in November 2021.

The Company plans to meet with the Agency in the coming weeks to align on the next steps to obtain approval.

“We remain committed to the hemophilia community and to leading the way to the first ever gene therapy in hemophilia A,” said Jean-Jacques Bienaim茅, Chairman and Chief Executive Officer of BioMarin.聽 “We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter.聽 We are confident in valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A.”

The application was based on the Phase 3 study interim analysis of study participants treated with investigational product manufactured by the to-be-commercialized process and three-year data from the Phase 1/2 Study.聽The FDA had granted valoctocogene roxaparvovec Priority Review status and Breakthrough Therapy and Orphan Drug designations.聽 Valoctocogene roxaparvovec maintains Breakthrough Therapy and Orphan Drug designations.

The ongoing valoctocogene roxaparvovec clinical trials will continue while BioMarin is exploring next steps to obtain approval. 聽The European Medicines Agency (EMA) review of the Company’s Marketing Authorization Application (MAA) for valoctocogene roxaparvovec is ongoing.

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