Exagamglogene autotemcel (exa-cel) was granted U.S. Food and Drug Administration (FDA) approval in January to treat transfusion-dependent beta thalassemia in patients age 12 or older.
Exa-cel is an ex vivo CRISPR/Cas9 gene-edited cell therapy that was approved for the treatment of sickle cell disease in December 2023.
Prior to approval, the application for this indication was granted both Fast Track and Regenerative Medicine Advanced Treatment designations. Common side effects include febrile neutropenia, mouth sores, and decreased appetite.
“Today’s approval is an important step in the advancement of an additional treatment option for individuals with beta thalassemia, a debilitating disease that places individuals at risk of many serious health problems,” said Nicole Verdun, MD, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “The approval of a cell-based gene therapy for this condition using CRISPR/Cas9 technology reflects FDA’s continued commitment to supporting safe and effective treatments that leverage the most promising and cutting-edge medical technologies.”
Health care professionals who administer the treatment are required to have experience in hematopoietic cell transplantation. As a result, Vertex Pharmaceuticals is establishing a system of nine authorized treatment centers across the country in partnership with CRISPR with plans to establish more.
Sources: CRISPR Therapeutics, January 16, 2024. FDA, January 16, 2024.
