BioMarin nets conditional approval for valrox in Europe, marking the first hemophilia gene therapy

As BioMarin prepares to head back to the FDA with its hemophilia A gene therapy, the EU is sailing forward at full speed ahead.

The European Commission granted conditional approval to the company’s valoctocogene roxaparvovec — more colloquially known as valrox — Wednesday afternoon, making it the first hemophilia A gene therapy approved anywhere. Valrox, to be branded on the continent as Roctavian, will treat adults with severe hemophilia A who need regular treatments to prevent bleeding episodes.