FDA Approves Vertex & CRISPR’s Gene Editing Therapy to treat Beta Thalassemia

Treat Beta Thalassemia

The FDA has approved Vertex and CRISPR’s gene-editing therapy Casgevy to treat beta thalassemia. Casgevy becomes the first therapy for the rare blood disorder to use the innovative CRISPR gene-editing technology.

Vertex Pharmaceuticals and CRISPR Therapeutics have scored an FDA approval for their gene-editing therapy Casgevy (exa-cel) to treat transfusion-dependent beta thalassemia (TDT). The approval comes less than six weeks after the U.S. regulator signed off on Casgevy to treat patients with sickle cell disease (SCD).

“On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” Reshma Kewalramani, M.D., Vertex’s CEO said in a release.

The companies have opened nine authorized treatment centers (ATCs) to administer Casgevy to patients, Vertex said. Each of the facilities can provide Casgevy in either indication.

Additional ATCs will be activated “in the coming weeks,” Vertex added.

This endorsement came two months earlier than predicted by the FDA!

Currently, the approval covers Transfusion Dependent Thalassemia patients ages 12 and older who need regular transfusions.

Vertex and CRISPR charge $2.2 million for the one-time treatment. A lifetime of healthcare costs in the U.S to manage TDT are estimated to exceed $5 million, the companies said.

Source: https://www.fiercepharma.com/pharma/vertex-crispr-win-early-fda-nod-gene-therapy-casgevy-treat-beta-thalassemia

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