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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Last year, the European Commission approved a record 55 therapies containing a new active substance, including 21 medicines for rare diseases. Among them were a number of gene therapies for indications such as cancer and hemophilia.
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.