The Food and Drug Administration (FDA) has approved the CRISPR gene editing-based therapy Exagamglogene Autotemcel, also known as Exa-Cel (Casgevy), by Vertex Pharmaceuticals for individuals 12 years and older with transfusion-dependent beta-thalassemia, a rare inherited blood disorder.
The approval, which came more than two months ahead of the scheduled effectiveness date of March 30th, is the second for the groundbreaking therapy. The FDA previously greenlit the CRISPR gene therapy for the treatment of sickle cell anemia last December.
Vertex and CRISPR Therapeutics‘ autologous, ex vivo gene-edited CRISPR/Cas9 therapy is the first to utilize the CRISPR gene editing tool.
The approval for transfusion-dependent beta-thalassemia is based on data from key studies showing a „consistent and sustained response to treatment“ in 52 patients who received an infusion and were observed for up to 4 years. According to a Vertex press release last year, the treatment conferred transfusion independence to patients with transfusion-dependent beta-thalassemia.
Vertex noted in a new press release that the expanded approval means approximately 1,000 patients aged 12 and older are eligible for this indication. Exa-Cel requires administration at authorized treatment centers with experience in stem cell transplantation.
The therapy, which has a list price of $2.2 million in the US, is expected to be initially available at nine authorized treatment centers at the beginning of the year, with more to follow.
Sharon Worcester, MA, is an award-winning medical journalist based in Birmingham, Alabama, who writes for Medscape, MDedge, and other partner sites. She is currently focusing on oncology but has also covered a wide range of other medical specialties and health topics. She can be reached at email@example.com or on Twitter: @SW_MedReporter.