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Gene Therapy: US FDA Labeling For Vertex’s Casgevy, Bluebird’s Lyfgenia Reflect Different Risks

 
• By Sue Sutter

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.

Apples and oranges
The first gene therapies for sickle cell disease have some substantial differences in safety labeling. • Source: Shutterstock

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 bio’s Lyfgenia (lovotibeglogene...

Key Takeaways

  • Bluebird's Lyfgenia gets a boxed warning on hematological malignancies resulting from two leukemia cases in the clinical program.

  • Labeling for Vertex's Casgevy warns about...

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