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Biomarin's previously rejected hemophilia drug gets special status at the FDA

The FDA in the US has given Biomarin's gene therapy against hemophilia special status, which will speed up the approval process. The treatment was rejected in August last year.

Photo: Manuel Balce Ceneta/AP/Ritzau Scanpix
BY ELIZABETH MØNSTED JOHANSEN, TRANSLATED BY NIELSINE NIELSEN

When Biomarin again tries to get the US Food and Drug Administration (FDA) to approve the hemophilia treatment valoctocogene roxaparvovec, it'll get some help along the way.

On Tuesday, the firm informs that the drug for treating hemophilia A has received a special status at the FDA known as regenerative medicine advanced therapy (RMAT).

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