1 July 2023 - After quite the regulatory process, with Roctavian (valoctocogene roxaparvovec) BioMarin has a potential blockbuster. On 29 June, Roctavian became the first gene therapy approved in the US in the haemophilia A space.
Listed at $2.9 million per single-use, Roctavian is also one of the most expensive therapies ever approved. Now the company turns to the difficult task of commercialising the product. Offering warranties may be a viable option.