Pharmaceutical Executive January 17, 2024
Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Cas9 therapy Casgevy approved as a one-time treatment for transfusion-dependent beta thalassemia on the heels of its approval last month for sickle cell disease.
The FDA has granted another approval to Vertex Pharmaceuticals’ and CRISPR Therapeutics’ gene-editing therapy Casgevy (exagamglogene autotemcel [exa-cel]) as a one-time treatment for transfusion-dependent beta thalassemia (TDT) in patients 12 years of age and older.1 Last month, Casgevy became the first FDA-approved novel genome editing technology for sickle cell disease (SCD), which was a significant advancement in gene therapy. The latest approval was months ahead of the FDA’s assigned Prescription Drug User Fee Act (PDUFA) date.
“We are pleased with the approval of Casgevy in TDT well ahead of the PDUFA...
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