Vertex Pharmaceuticals Says FDA Approved Casgevy for TDT Patients
January 16 2024 - 2:07PM
Dow Jones News
By Will Feuer
Vertex Pharmaceuticals said the U.S. Food and Drug
Administration has approved its Casgevy drug for the treatment of
transfusion-dependent beta thalassemia in patients 12 years and
older.
Transfusion-dependent beta thalassemia is a serious,
life-threatening genetic disease. Patients require lifelong
treatment and significant use of health care resources, Vertex
said, and the disease ultimately results in reduced life expectancy
and a lower quality of life. In the U.S., the median age of death
for patients living with TDT is 37 years, Vertex said.
Casgevy is a Crispr/Cas9 gene-edited cell therapy. The FDA has
already approved it for sickle cell disease. It is the world's
first medicine employing Crispr technology, a Nobel Prize-winning
discovery that promised a powerful new tool for modifying genes to
treat disease and improve crop production.
Write to Will Feuer at Will.Feuer@wsj.com
(END) Dow Jones Newswires
January 16, 2024 14:52 ET (19:52 GMT)
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