First FDA-approved CRISPR-based gene therapy cleared for 2nd indication

Cell Gene Therapy Dna Social

A month after the U.S. Food and Drug Administration (FDA) approved Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy for the treatment of vaso-occlusive crises in patients with sickle cell disease (SCD), the agency has given it a second U.S. regulatory approval ahead of schedule.

The FDA approved the CRISPR-based gene-editing therapy Casgevy (exagamglogene autotemcel) for the treatment of transfusion-dependent β-thalassemia (TDT) in patients 12 years and older on Tuesday.

TDT is a form of thalassemia, an inherited blood disorder in which the production of hemoglobin is impaired. TDT requires regular blood transfusions and can be life-threatening.

In a statement, Vertex and CRISPR Pharmaceuticals said that the anticipated approval date for the use of Casgevy for TDT was March 30.

“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,” Dr. Reshma Kewalramani, CEO and president of Vertex, said. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing Casgevy to eligible patients who are waiting.”

Casgevy was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. for both SCD and TDT in November.

Because the administration of Casgevy requires experience in stem cell transplantation, Vertex said that it is working with hospitals to establish a network of independently operated authorized treatment centers (ATCs) throughout the U.S. to administer Casgevy to eligible patients with SCD and TDT. There are currently nine active ATCs in the U.S.; the firm says that additional ATCs will be opened in the coming weeks. A complete list of ATCs can be accessed at

Page 1 of 2
Next Page