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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