Health and Healthcare

What This FDA Response Means for BioMarin's Gene Therapy for Hemophilia

Chris Lange

Published: August 19, 2020 9:55 am

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) stock was crushed on Wednesday after the company announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to its Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A.

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.

According to the CRL, the FDA introduced a new recommendation for two years of data from the company’s ongoing 270-301 Phase 3 study to provide substantial evidence of a durable effect using annualized bleeding rate as the primary endpoint.

Looking forward, the FDA recommended that BioMarin complete its the Phase 3 Study and submit two-year follow-up safety and efficacy data on all study participants. The 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.

The Phase 3 study was fully enrolled in November 2019, and the last patient will complete two years of follow up in November 2021.

It’s worth noting that the BLA 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.

Previously, the FDA had granted valoctocogene roxaparvovec Priority Review status and Breakthrough Therapy and Orphan Drug designations. Valoctocogene roxaparvovec maintains its Breakthrough Therapy and Orphan Drug designations despite the CRL.

BioMarin stock traded down about 30% to $83.10 Wednesday morning, in a 52-week range of $62.88 to $131.95. The consensus price target is $136.91.