Vertex's Casgevy gene therapy receives FDA approval for children with sickle cell disease and beta-thalassemia

Vertex announced that its gene therapy Casgevy has been granted FDA approval for use in pediatric patients. The decision expands the therapy's reach beyond adult indications.

Casgevy is now authorized to treat children with sickle cell disease who experience recurrent vaso-occlusive crises, as well as those with transfusion‑dependent beta‑thalassemia. This marks a significant step for gene‑based treatments in these blood disorders.

In a separate development, United Therapeutics reported the acquisition of a biotech startup, underscoring continued consolidation activity in the sector.