(Reuters) – The U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals and CRISPR Therapeutics’ gene therapy to treat a rare blood disorder requiring regular blood transfusions, in patients 12 years and older, Vertex said on Tuesday.
The therapy, branded as Casgevy, was approved for transfusion-dependent beta thalassemia and is based on the Nobel Prize-winning CRISPR gene editing technology.
The decision earns Casgevy the second approval in the United States after the health regulator, in December, greenlighted it for sickle cell disease, another inherited blood disorder.
Casgevy requires administration through authorized treatment centers with experience in stem cell transplantation, Vertex said.
(Reporting by Mariam Sunny in Bengaluru; Editing by Shailesh Kuber)
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