FDA Approves Vertex, CRISPR’s Casgevy to Treat Beta Thalassemia

Pictured: FDA headquarters/iStock, Grandbrothers

Pictured: FDA headquarters/iStock, Grandbrothers

The FDA on Tuesday approved Vertex Pharmaceuticals and partner CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) Cas9 gene-edited cell therapy as a one-time treatment for transfusion-dependent beta thalassemia patients 12 years of age and older.

The announcement comes just a month after Casgey was approved by the FDA in December 2023 to treat patients with sickle cell disease, when it became the first CRISPR gene editing therapy to reach the U.S. market. The regulator’s decision on Tuesday to expand the use of Casgevy for the treatment of transfusion-dependent beta thalassemia (TDT) was more than two months ahead of its PDUFA date.

Vertex and CRISPR Therapeutics expected an FDA decision for Casgevy in TDT by March 30, 2024.

“On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to secure approval for TDT now well ahead of the PDUFA date,” Vertex CEO Reshma Kewalramani said in a statement. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing Casgevy to eligible waiting patients.”

Vertex is currently working with hospitals to establish a network of authorized treatment centers (ATCs) to offer the therapy to patients, with more coming online “in the coming weeks,” according to the company’s announcement. So far, nine ATCs across the country can offer Casgevy to patients with TDT and sickle cell disease. The company noted that “the administration of Casgevy requires experience in stem cell transplantation” and that around 1,000 patients in the U.S. are now eligible for the one-time treatment.

Casgevy leverages the Nobel prize-winning technology CRISPR/Cas9 gene editing system, discovered more than a decade ago by Jennifer Doudna and Emmanuelle Charpentier, to modify patients’ hematopoietic (blood) stem cells.

CRISPR Therapeutics CEO Samarth Kulkarni said in a separate announcement that Tuesday’s FDA approval of Casgevy “is a reflection of the power and versatility of the CRISPR platform to bring a potentially curative treatment option to patients suffering from this devastating disease.”

Casgevy’s FDA approval for sickle cell disease (SCD) last month came the same day as bluebird bio’s cell-based gene therapy Lyfgenia, which uses a lentiviral vector (gene delivery vehicle) for genetic modification and was also approved for SCD. However, Vertex and CRISPR appear to have the edge over bluebird, given that the list price for Casgevy is $2.2 million per patient compared to $3.1 million for Lyfgenia. In addition, Lyfgenia has a black-box warning related to certain cases of blood cancer in patients treated with the therapy, while Casgevy does not.

Vertex did not provide a list price for Casgevy for the treatment of TDT in Tuesday’s announcement. Bluebird bio’s gene therapy Zynteglo, which was approved by the FDA to treat beta-thalassemia in August 2022, costs $2.8 million.

Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.

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