Casgevy (exagamglogene autotemcel)
- Region
- 🇪🇺 EU
- Active substance
- exagamglogene autotemcel
- Company
- Vertex Pharmaceuticals (Ireland) Limited
- Status
- Authorised
- Therapeutic area
- beta-Thalassemia; Anemia, Sickle Cell
- CHMP opinion
- Thu 14 Dec 2023
- EU approval
- Fri 09 Feb 2024
- Designations
- Orphan, PRIME, ATMP, Conditional, Additional monitoring
- Application
- EMEA/H/C/005763
- ATC
- B06AX05
- Official record
- www.ema.europa.eu
Indication
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.
Regulatory milestones
No milestones recorded in the last year.
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.