Vertex Pharmaceuticals Gets FDA OK for Sickle Cell Disease Treatment in Young Children

By Elias Schisgall

The Food and Drug Administration approved the use of Vertex Pharmaceuticals's sickle cell disease treatment, Casgevy, for patients as young as two years old.

The agency said Wednesday that it issued a supplemental approval for the drug, known generically as exagamglogene autotemcel, for patients between the ages of two and 11. It was previously approved to treat patients 12 years of age and older.

Casgevy is approved to treat patients with either sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β thalassemia, another blood disorder. The FDA said Casgevy is the first gene therapy approved for sickle cell disease patients as young as two.

The approval is the eighth selected for the FDA Commission's National Priority Voucherpilot program, the agency said.

Write to Elias Schisgall at elias.schisgall@wsj.com