FDA approves Casgevy (exagamglogene autotemcel) for sickle cell disease, first CRISPR-based gene therapy in the US

CRISPR Therapeutics

The FDA approved Casgevy, the world's first CRISPR-based gene-editing therapy, for sickle cell disease in patients 12 years and older. Among 44 patients treated, 31 had sufficient follow-up data, and 29 (93.5%) achieved at least 12 consecutive months free from severe vaso-occlusive crises. Vertex set the wholesale acquisition cost at $2.2 million.

• 93.5% of evaluable patients achieved freedom from severe vaso-occlusive crises for at least 12 months
• One-time autologous cell therapy edits patient's own blood stem cells ex vivo
• Approximately 16,000 US patients with sickle cell disease may be eligible
• European approval granted February 2024; beta-thalassemia approval granted January 16, 2024