BioMarin Hopes A Single Warranty Will Make Gene Therapy Reimbursement Simpler

Firm Is Offering Same Outcomes-Based Deal To All Payers

 
• By Jessica Merrill
  
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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.

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Roctavian Is Slated To Launch In Europe And The US This Year

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. Morgan Healthcare...

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Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area. 

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