The approval, which comes more than 2 months ahead of a target action date of March 30, marks the second for the landmark therapy. The FDA greenlit the CRISPR gene therapy to treat sickle cell disease last December.
The autologous, ex vivo, CRISPR/Cas9 gene-edited therapy from Vertex and CRISPR Therapeutics is the first to use the gene-editing tool CRISPR.
The transfusion-dependent beta thalassemia approval is based on data from pivotal studies showing “consistent and durable response to treatment” in 52 patients who received an infusion and followed for up to 4 years. Treatment conferred transfusion independence in patients with transfusion-dependent beta thalassemia, according to a press release from Vertex late last year.
Vertex noted in a new press statement that expanded approval means about 1000 patients aged 12 years or older will be eligible for the one-time treatment for this indication.
Exa-cel requires administration at authorized treatment centers experienced in stem cell transplantation.
The therapy, which has a list price of $2.2 million in the United States, should be available initially at nine authorized treatment centers early this year, with more to come, according to Vertex.
A version of this article appeared on Medscape.com.