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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Orphans Account For More Than A Third Of EU New Drug Approvals In 2022
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.
BioMarin Hopes A Single Warranty Will Make Gene Therapy Reimbursement Simpler
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.
CSL’s Hemgenix Gene Therapy Does Not Need A REMS, FDA Tells National Hemophilia Foundation
Peter Marks says labeling for CSL’s hemophilia B treatment adequately conveys its risks. He also denies NHF request that the label cite eligibility criteria used in clinical trials, responding to a citizen petition on the day the agency approves the therapy.