CRISPR, Vertex Get FDA OK of Casgevy Gene Therapy in Sickle-Cell Disease
08 Dezembro 2023 - 1:46PM
Dow Jones News
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)
Copyright (c) 2023 Dow Jones & Company, Inc.
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