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🏅 FDA Orphan Designation

CASGEVY

EXAGAMGLOGENE AUTOTEMCEL

Manufacturer: Vertex Pharmaceuticals Incorporated

Indicated for:
Sickle cell diseaseOrphanBeta-thalassemia major

FDA-Approved Indications (2)

Sickle cell diseaseOrphan Designation

1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises transfusion-dependent β - thala…

indicated for the treatment of patients aged 12 years and older with transfusion-dependent β-thalassemia (TDT)

Population: pediatric ≥12 years and older

Indications & Usage

1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises transfusion-dependent β - thalassemia (TDT) CASGEVY is an autologous genome edited hematopoietic stem cell-based gene therapy indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). ( 1 ) transfusion-dependent β-thalassemia (TDT). ( 1 )

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CASGEVY
Vertex Pharmaceuticals Incorporated
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Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.