# FDA Grants First Gene Therapy Approval for Children as Young as 2 with Sickle Cell Disease

> fda · REPORTED · 2026-07-01

On July 1, 2026, the FDA issued a supplemental approval for Casgevy (exagamglogene autotemcel) expanding its label to patients aged 2 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia, making it the first gene therapy approved for this younger pediatric population.

- Company: Vertex Pharmaceuticals / CRISPR Therapeutics (Casgevy)
- Sector: therapeutics
- Primary source: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-young-children-sickle-cell-disease

---
Source: MedFuel Intel news monitor. Verification tier: REPORTED. Informational only, not investment advice. Corrections: media@medfuelinc.com.