FDA Advisory Committee concludes review on exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease
CRISPR Therapeutics announced the completion of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee meeting for exagamglogene autotemcel (exa-cel) for the treatment of SCD in people ages 12 and older with recurrent vaso-occlusive crises (VOCs)
Exa-cel is the first potential therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals.
Ultimately, the panel appeared convinced that Vertex Pharmaceuticals, which developed the sickle cell treatment, had done enough to show the therapy is safe, although they pointed to several avenues for further study.
Instead, the focus was on CRISPR, the technology and esoteric questions of how exactly companies like Vertex can be sure its medicine is not making wayward gene edits. Panelists appeared unsure how strictly to set the standard, and wary of asking for more data than was actually useful.
If approved, exa-cel could be the first genetic therapy available to approximately twenty thousand people with severe SCD in the U.S. The FDA granted priority review for exa-cel in the treatment of people with SCD and assigned a Prescription Drug User Fee Act (PDUFA) action date of December 8, 2023. Exa-cel’s Biologics License Application (BLA) for transfusion-dependent beta-thalassemia (TDT) was assigned a PDUFA date of March 30, 2024.
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