The Food and Drug Administration on Tuesday approved a CRISPR-based medicine to treat beta thalassemia, an inherited blood disorder. It’s the same potentially curative therapy cleared by the FDA in December to treat sickle cell disease.
The expanded approval of the therapy, called Casgevy, was widely expected but came two months ahead of the FDA’s decision deadline, known as a PDUFA date. The therapy was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
“On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to now secure approval for thalassemia well ahead of the PDUFA date,” Vertex CEO Reshma Kewalramani said in a statement. “Thalassemia patients deserve new, potentially curative treatment options, and we look forward to bringing Casgevy to eligible patients who are waiting.”