BioMarin Hopes A Single Warranty Will Make Gene Therapy Reimbursement Simpler
Firm Is Offering Same Outcomes-Based Deal To All Payers
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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Any given year sees a variety of clinical trials produce positive or negative results. But some of those trials are especially important either for moving a field forward or for a company’s overall development and commercial strategy. Here, Scrip looks at some of the more notable trial successes of the last year.
Daily notebook from the J.P. Morgan Healthcare Conference: Gene therapy Vertex enters pricing negotiations on gene therapy, Sanofi sees opportunity in hemophilia despite new gene therapy, and AbbVie raises guidance ahead of the introduction of Humira biosimilars. Plus ICER's Steven Pearson and former Biogen CEO George Scangos weigh in on Alzheimer's developments.
FDA review of the company's BLA for a hemophilia gene therapy could be delayed by three months to allow the agency to review a three-year data analysis from an ongoing trial.