Casgevy
What Casgevy is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
- PRIME: a priority medicine that the EMA supported early because it may meet an unmet need.
- Conditional approval: approved on less complete data because the need is high; more data must follow.
Key facts
- Brand
- Casgevy
- Active substance
- exagamglogene autotemcel
- Disease area
- beta-Thalassemia; Anemia, Sickle Cell
- Medicine group
- Other hematological agents
- ATC code
- B06AX05
- Marketing authorisation holder
- Vertex Pharmaceuticals (Ireland) Limited
- Authorised in the EU since
- 2024
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Casgevy used for?
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 …
What is the active substance in Casgevy?
Casgevy contains exagamglogene autotemcel.
Who makes Casgevy?
The EU marketing authorisation holder is Vertex Pharmaceuticals (Ireland) Limited.
Is Casgevy an orphan medicine?
Yes. Casgevy is designated an orphan medicine in the EU, for a rare disease (beta-Thalassemia; Anemia, Sickle Cell).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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