FDA approves Vertex and CRISPR’s Casgevy as first CRISPR-edited therapy for beta-thalassemia

The FDA has ap­proved Ver­tex Phar­ma­ceu­ti­cals and CRISPR Ther­a­peu­tics’ Cas­gevy to treat be­ta-tha­lassemia, Ver­tex said Tues­day, mak­ing it the first-ever CRISPR-edit­ed ther­a­py for the rare ge­net­ic blood dis­or­der.

In De­cem­ber, the agency OK’d Cas­gevy to treat se­vere sick­le cell dis­ease. Ver­tex and CRISPR co-de­vel­oped Cas­gevy, which they priced at $2.2 mil­lion for the one-time ex vi­vo treat­ment.

The be­ta-tha­lassemia ap­proval is for pa­tients 12 years and old­er who need reg­u­lar blood trans­fu­sions to sur­vive. Sim­i­lar to the sick­le cell mar­ket, Cas­gevy will com­pete with a gene-edit­ed ther­a­py from the start­up blue­bird bio. Blue­bird’s Zyn­te­glo was ap­proved in 2022 to treat be­ta-tha­lassemia.

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