CRISPR, Vertex Get FDA OK of Casgevy Gene Therapy in Sickle-Cell Disease

By Colin Kellaher

CRISPR Therapeutics and Vertex Pharmaceuticals have won Food and Drug Administration approval of their Casgevy gene therapy for sickle-cell disease, marking the first U.S. green light for a drug using the gene-editing technology known as Crispr.

The FDA on Friday said the approval covers Casgevy for the treatment of sickle-cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises

U.K. regulators last month gave the world's first approval of Casgevy, granting conditional marketing authorization for the treatment of sickle-cell disease and transfusion-dependent beta thalassemia, a pair of inherited blood disorders.

An FDA decision on Casgevy in transfusion-dependent beta thalassemia is due by the end of March.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

December 08, 2023 11:31 ET (16:31 GMT)

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