BioMarin Gears Up To Give Roctavian Another Go

Voxzogo Sales Could Exceed $100m

The company is aiming for an outcomes-based payment model in the US, while it is confident the gene therapy will fare better than some others have in Europe.

3-D Rendering Red Blood Cells — BioMarin is preparing to seek approval again for Roctavian in hemophilia A • Source: Shutterstock

Less than a year and a half after it received a surprise complete response letter (CRL) from the US Food and Drug Administration for Roctavian (valoctocogene roxaparvovec) in severe hemophilia A, BioMarin Pharmaceutical Inc. is now ready to reapproach regulators in the US as it awaits a decision on the gene therapy from European regulators and gets a sense of potential 2022 sales of Voxzogo (vosoritide), its recently approved drug for achondroplasia.

CEO Jean-Jacques Bienaimé provided the outlook for 2022 at BioMarin on 10 January at the J.P. Morgan Healthcare Conference.

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