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FDA Grants Additional Approval for Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy

NextEdge Admin

26 Dec 2023

2 min 35 sec

The US Food and Drug Administration (FDA) has granted a second approval for Vertex Pharmaceuticals’ CRISPR gene-editing therapy, Casgevy (exagamglogene autotemcel), expanding its use to treat transfusion-dependent beta thalassemia (TDT). Casgevy, developed by Vertex in collaboration with CRISPR Therapeutics, received initial approval for sickle cell disease treatment in December. The UK Medicines and Healthcare Products Regulatory Agency (MHRA) had also granted conditional marketing authorization for the drug in both disease settings in November.

The FDA’s decision on Casgevy’s TDT indication was anticipated by March 30, but the approval came earlier. Vertex’s President and CEO, Reshma Kewalramani, expressed excitement over securing approval for TDT ahead of schedule, following the historic FDA approval for sickle cell disease.

Casgevy is approved as a one-time, potentially curative treatment for patients aged 12 and older with TDT, a genetic blood disorder resulting in insufficient hemoglobin production. The therapy involves ex vivo editing of the BCL11A gene in the patients’ CD34-positive hematopoietic stem and progenitor cells using CRISPR-Cas9. This editing is designed to boost fetal hemoglobin production, aiding oxygen delivery similar to the adult version.

Interim data from pivotal trials submitted to the FDA demonstrated that 24 out of 27 TDT patients achieved transfusion independence for at least 12 months post-treatment. Additional findings from an ongoing Phase III trial presented at the American Society of Hematology’s annual meeting showed 32 of 35 patients achieving transfusion independence after 16 months of follow-up. Vertex set a wholesale acquisition cost of $2.2 million for Casgevy in the US when announcing its approval for sickle cell disease in December.