Vertex, CRISPR's Casgevy gets FDA nod for beta thalassemia

Jan. 17, 2024

Vertex Pharmaceuticals and CRISPR Therapeutics announced that the U.S. FDA has approved their CRISPR/Cas9 genome-edited cell therapy, Casgevy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

The approval for TDT, a rare blood disorder that leads to chronic anemia, comes far ahead of the FDA's PDUFA target action date of March 30, 2024. Casgevy notched its first U.S. approval back on December 8, when it became the first gene-edited cell therapy to treat patients with sickle cell disease.

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) was the first in the world to sign off on Casgevy, granting approval for both indications back in mid-November.

The administration of the drug, which is the first therapeutic product arising from a 2015 collaboration between CRISPR Therapeutics and Vertex, requires experience in stem cell transplantation. According to the partners, Vertex is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. There are currently only 9 ATCs in the U.S. that can administer Casgevy, but Vertex says it is expanding the list.