Although the US Food and Drug Administration approved the first two gene therapies for sickle cell disease in a sweeping same-day action, product labeling reflects potential risks specific to each therapy's mechanism of action and clinical program to date.
On 8 December, the FDA approved Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG’s Casgevy (exagamglogene autotemcel, or exa-cel) on the user fee goal date, and bluebird
Key Takeaways
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Bluebird's Lyfgenia gets a boxed warning on hematological malignancies resulting from two leukemia cases in the clinical program.
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Labeling for Vertex's Casgevy warns about potential for off-target effects, and the sponsor must conduct more preclinical analyses of these risks postapproval