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BioMarin Hopes A Single Warranty Will Make Gene Therapy Reimbursement Simpler

Firm Is Offering Same Outcomes-Based Deal To All Payers

Executive Summary

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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Five Clinical Trial Hits Of 2022

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.

J.P. Morgan Day Three: 2023 Is A Year Of Commercial Challenges

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. 

BioMarin Prepares Investors For A Possible US Hemophilia Gene Therapy Delay

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.

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