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.
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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.
San Rocco, whose potential rival gene therapy for thalassemia is in preclinical development, had wanted FDA to delay approval of bluebird’s BLA until resolution of a patent case and a ‘careful investigation’ of Zynteglo’s risks.
The approval is a breakthrough for BioMarin, which is hoping to seize market share from Factor VIII replacement therapies and Roche’s Hemlibra.