The management team talked to Scrip at J.P. Morgan about plans for launching the first gene therapy for hemophilia A in Europe and the US, and its unique approach to reimbursement.
BioMarin Pharmaceutical Inc. is gearing up for a major commercial challenge as it launches the first gene therapy for hemophilia A, but it is preparing by setting a new model for outcomes-based reimbursement. Roctavian (valoctocogene roxaparvovec) has already been approved in Europe, and in the US it is pending at the US Food and Drug Administration with a 31 March user fee date.
CEO Jean Jacques Bienaimé and other members of the management team talked to Scrip ahead of the J.P
Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.
Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.
The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.
Adstiladrin sales hit €70m in first full year on the market
Sales of the company’s approved cancer therapies were weaker or down, but Trodelvy is likely to see a label expansion and cell therapies are making progress.
BriaCell announced its cell therapy Bria-IMT plus checkpoint inhibitors bested Gilead’s Trodelvy in a Phase II trial in HR+ breast cancer; a Phase III trial is enrolling.
The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.
