Phase 3
Development Phase
1
Upcoming Catalysts
0
Historical Events
1
Regulatory Approvals
5 competitors in Sickle Cell Disease
View Full LandscapeTarget Indication
Sickle Cell Disease
Mechanism of Action
Gene Editing (CRISPR)
Clinical Trial
NCT03745287Last updated: 12/3/2025
No patent data available. Pre-approval drugs may not have Orange Book listings.
Casgevy
β thalassemia Casgevy is indicated for the treatment of transfusion dependent β thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is not available. Sickle cell disease Casgevy is indicated for the treatment of severe sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso occlusive crises (VOCs) for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is not available.