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.
Gene Therapy: US FDA Labeling For Vertex’s Casgevy, Bluebird’s Lyfgenia Reflect Different Risks
Agency grants same-day approval to the first two gene therapies for sickle cell disease; bluebird’s lentiviral-based lovo-cel carries a boxed label warning on hematologic malignancies, while Vertex’s CRISPR-Cas9 exa-cel carries a warning and precaution about potential off-target effects. Only the Vertex product qualified for a rare pediatric disease priority review voucher.
