FDA Approves Vertex/CRISPR Gene Therapy for Beta Thalassemia

Casgevy, the first CRISPR-based treatment for TDT, to be available early this year at $2.2 million

FDA approves Vertex/CRISPR gene therapy, Casgevy, for transfusion-dependent beta thalassemia (TDT), an inherited blood disorder, in patients 12 years and older.
Casgevy becomes the first treatment based on CRISPR gene editing technology to secure approval for TDT in the United States.
The therapy, which requires administration through authorized treatment centers with experience in stem cell transplantation, will be available early this year at a list price of $2.2 million in the United States.
More than 100,000 people are estimated to have TDT globally, with at least 1,200 people with the disorder in the United States.
The approval comes two months ahead of the FDA's expected action date of March 30.