FDA Rejects Approval of BioMarin's Gene Therapy Product for Hemophilia A


BioMarin Gene Therapy Manufacturing Facility

Today was a big blow for BioMarin and its gene therapy product, valoctocogene roxaparvovec for severe hemophilia A

BioMarin announced that the FDA issued a Complete Review Letter (CRL) to indicate that the application is not ready for approval in its present form. The FDA introduced a new recommendation for two years of data from the ongoing Phase 3 study to provide substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint. The Phase 3 study was fully enrolled in November 2019, and the last patient will complete two years of follow up in November 2021.


So, if the FDA is happy with the Phase 3 data, then BioMarin can bring this to market. However, with this set-back, that will not be until 2022.


We have mentioned in the past that there has been some concerning data regarding factor levels plateauing in patients in year three after receiving the gene therapy. HemoAware's Founder, Jesse John Francis Clark, went to Dallas last year to participate in BioMarin roundtable regarding the marketing of this gene therapy. BioMarin already had all of its marketing materials ready and was anticipating this product hitting the market by this fall.


Well, that isn’t happening and it’s a tough loss for BioMarin. There are currently two other big gene therapy pharma companies trying to beat BioMarin in the gene therapy market. Spark Therapeutics has its SPK-8011 gene therapy which Phase 3 will start in 2021 and Sangamo Therapeutics has SB-525 which Phase 3 is currently ongoing.


We will keep you posted if any of these products get FDA approval.


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