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

by mikkel aabenhus hemmingsen, translated by daniel pedersen

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.

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Biomarin's hemophilia drug meets all endpoints in phase III study
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Sobi receives FDA breakthrough designation for hemophilia drug
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