Vertex Pharmaceuticals Says FDA Approved Casgevy for TDT Patients

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 [e-mail]

(END) Dow Jones Newswires

January 16, 2024 14:52 ET (19:52 GMT)

Copyright (c) 2024 Dow Jones & Company, Inc.