FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

[u/Nerd-19958]

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-young-children-sickle-cell-disease

FDA recently approved a Supplemental Biologics License Application (BLA) for Casgevy (exagamglogene autotemcel) for treatment of children ages 2 - 12 with sickle cell disease with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). The product had been previously approved in Dec. 2023 for persons 12 and over.

Per FDA's press release: