![]() ![]() ![]() Per the company, a tentative approval will make beti-cel the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia in the United States. Β-thalassemia is a severe genetic disease for those requiring regular RBC transfusions caused by mutations in the β-globin gene, which may cause significantly reduced adult hemoglobin (Hb). The regulatory body has set a target action date of May 20, 2022. The BLA is seeking approval of beti-cel, a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. ( BLUE Quick Quote BLUE - Free Report) recently announced that the FDA has accepted the biologics license application (BLA) for betibeglogene autotemcel (beti-cel) for priority review.
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