
At a US Food and Drug Administration (FDA) meeting on October 31, 2023, the panel members of the 76th Cellular, Tissue, and Gene Therapies Advisory Committee suggested that exagamglogene autotemcel (exa-cel), a groundbreaking therapy for severe sickle cell disease (SCD) utilizing CRISPR gene-editing technology, was safe enough for clinical use.
Exa-cel may be the first potentially curative treatment for SCD, a condition with significant, lifelong, and often fatal complications. The one-time therapy modifies the BCL11A gene in bone marrow stem cells to allow the production of fetal hemoglobin in adults in order to mitigate the impact of the diseases’ eponymous sickle-shaped blood cells.
Genetic Therapy with Potential to Cure SCD Goes to FDA for Approval
Exa-cel was developed in a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics. Stephanie Krogmeier, PhD, Vice President of Global Regulatory Affairs at Vertex Pharmaceuticals, proposed that exa-cel receive an indication “for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises” in a presentation to the FDA.
Generally, the FDA follows the nonbinding recommendations given by the panels of independent experts who comprise the advisory committees. In this case, the committee’s endorsement is likely to lead to FDA approval for exa-cel on December 8. If approved, exa-cel would be the first therapy to treat a genetic disease using CRISPR gene-editing technology.
In June 2023, the FDA accepted the Biologics License Application for exa-cel for the treatment of severe SCD and transfusion-dependent β-thalassemia and granted the treatment Priority Review status.
Reference
- Cellular, Tissue, and Gene Therapies Advisory Committee October 31, 2023 meeting announcement. FDA. October 31, 2023. Accessed November 1, 2023. https://www.fda.gov/advisory-committees/advisory-committee-calendar/cellular-tissue-and-gene-therapies-advisory-committee-october-31-2023-meeting-announcement-10312023#event-materials
Related: Stem Cell Gene Therapy Shows Promise as Treatment for Sickle Cell Disease