Hemophilia Gene Therapies Should Have REMS For Approval, National Hemophilia Foundation Says
Agency should require physician training, facility certification, and patient registry as a condition for approving BioMarin's and CSL Behring’s investigational gene therapies, the advocacy group says. It calls for treatment to be limited to the population studied with no-off label use.
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Running multiple product-specific postmarket trials for 15 years or longer would not be cost-effective and one industry stakeholder suggests workstreams eventually could merge.
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.
Sanofi/Sobi’s efanesoctocog is in line for US FDA action three months after CSL/uniQure’s gene therapy user fee goal, while BioMarin’s Roctavian resubmission and Novo Nordisk’s concizumab are waiting in the wings.