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Additional data demands delay Biomarin's hemophilia drug submission in US

The US health authority has requested more data pertaining to the company’s hemophilia A treatment candidate, and Biomarin has subsequently postponed its marketing approval submission.

Photo: Manuel Balce Ceneta/AP/Ritzau Scanpix

Gene therapy valoctocogene roxaparvovec will not be up for re-evaluation by the US Food and Drug Administration (FDA) until September, biotech firm Biomarin has announced in a press release.

In the same statement, Biomarin also reports that no patient who received the highest drug dose has required the preventive hemophilia treatment known as prophylactic Factor VIII in the six years since they started the firm’s gene therapy.

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