Casgevy cleared for pediatric sickle cell disease and β-thalassemia
- Sponsor
- Vertex Pharmaceuticals $VRTX
- Drug
- Casgevy · exagamglogene autotemcel
- Indication
- Sickle cell disease with recurrent vaso-occlusive crises; transfusion-dependent β-thalassemia — patients aged 2 years and older
FDA issued a supplemental approval expanding Casgevy (exagamglogene autotemcel) to patients aged 2 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia. Casgevy was previously approved for ages 12+, and this becomes the first gene therapy approved for patients as young as 2 with SCD.
Read original at fda.gov ↗From the source
FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease
For Immediate Release: July 01, 2026
The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β-thalassemia (TDT). This is the first gene therapy approved for patients aged 2 years and older with SCD.
Casgevy has been previously approved for the treatment of patients aged 12 years and older with SCD with recurrent VOCs or TDT.
"With today's decision, pediatric patients as young as 2 years of age can now access a critical additional treatment option to treat these debilitating, life-threatening diseases," said Karim Mikhail, Acting Director of the Center for Biologics Evaluation and Research (CBER).
Reproduced from FDA Press Releases · view original · public domain or open-licensed government work