Casgevy (exagamglogene autotemcel) · Vertex Pharmaceuticals
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). transfusion-dependent β-thalassemia (TDT).
Details
- Status
- Prescription
- First Approved
- 2023-12-07
- Patent Cliff
- 2031
- Routes
- Intravenous
- Dosage Forms
- Suspension
Casgevy Approval History
What Casgevy Treats
1 FDA approvalsOriginally approved for its first indication in 2023 .
- Other (1)
Other
(1 approval)- • Approved indication (Dec 2023)
Vertex Pharmaceuticals's other novel FDA approvals
Other CDER-designated drugs from the same sponsor (2016–2025).
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
Casgevy FDA Label Details
Indications & 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). transfusion-dependent β-thalassemia (TDT).
Read on TheraRadar
Casgevy Doesn’t Fix the Sickle Cell Mutation
It goes around it. That’s why it works.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.