Vertex reports FDA approves Casgevy for transfusion-dependent beta thalassemia

Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration has approved Casgevy, a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia in patients 12 years and older. “On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date. TDT patients deserve new, potentially curative treatment options, and we look forward to bringing CASGEVY to eligible patients who are waiting,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.