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# Vertex Pharma Gets 2nd OK for Gene Therapy
- URL: https://www.fdaweb.com/vertex-pharma-gets-2nd-ok-for-gene-therapy/
- Published: 2024-01-16T12:00:00.000Z
- Updated: 2026-09-14T14:20:24.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5156140

FDA has approved a Vertex Pharmaceuticals BLA for Casgevy (exagamglogene autotemcel \[exa-cel\]), a CRISPR/Cas9 gene-edited cell therapy for treating transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. This is the second approval granted for Casgevy; the first approval was granted 12/9/2023 for treating sickle cell disease ([see story](https://www.fdaweb.com/first-sickel-cell-gene-therapies-approved/)).

The company says that because administering Casgevy requires experience in stem cell transplantation, it is working with experienced hospitals to establish a network of authorized treatment centers (ATCs). Currently, nine such ATCs are able to offer the cell therapy to eligible patients with TDT and sickle cell disease.

TDT is described as a serious, life-threatening genetic disease that requires frequent blood transfusions and iron chelation therapy throughout a person’s life. “TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity,” the company says.