BioMarin aims to ‘guarantee’ hemophilia gene therapy’s efficacy in EU deals

Upon the European approval of gene therapy Roctavian to treat hemophilia A, BioMarin said it is negotiating with health authorities on a proposed net price of about €1.5 million, with an outcomes-based plan that would provide rebates if the treatment’s effect wanes within a window of several years.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said late Wednesday that the European Commission conditionally approved Roctavian valoctocogene roxaparvovec, a single-administration gene therapy that uses an adeno-associated viral serotype 5 (AAV5) vector to deliver the Factor VIII gene. The indication covers adults with severe hemophilia A without a history of Factor VIII inhibitors to counteract the effects of less permanent Factor VIII therapy...