Casgevy receives supplementary approval under the Commissioner's National Priority Voucher program

The FDA issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β thalassemia (TDT).

Casgevy is a one-time single gene therapy consisting of the patient’s own hematopoietic stem cells.

Casgevy was previously approved for the treatment of patients aged 12 years and older with SCD with recurrent VOCs or TDT. Based on product characteristics and clinical study data, extrapolation to the younger pediatric age population was granted to expand the indication to 2 years of age and above for both conditions.

The approval decision was granted 53 days after filing and represented the eighth approval selected for the Commissioner's National Priority Voucher pilot program.‍

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Source: Press Release