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Exagamglogene Autotemcel

Casgevy · ATC code B06AX05

Also known as: Exagamglogen-Autotemcel

Key facts
Active substance
Exagamglogene Autotemcel
ATC code
B06AX05
Authorisation
centrally authorised across the EU (EMA)
Dosage forms
Casgevy 4 - 13 × 106 cells/mL dispersion for infusion
Marketing authorisation holder
Vertex Pharmaceuticals (Ireland) Limited
Information as of
2026-10-27

Key facts

Exagamglogene autotemcel is an autologous CD34+ stem cell therapy genetically modified with CRISPR/Cas9. It is used for transfusion-dependent β-thalassemia and severe sickle cell disease from 12 years of age.

What it is used for

  • Transfusion-dependent β-thalassemia (TDT) in patients 12 years and older for whom stem cell transplantation is appropriate and no HLA-matched related donor is available
  • Severe sickle cell disease with recurrent vaso-occlusive crises in patients 12 years and older, under the same conditions

Dosing

  • Single infusion in an authorised treatment centre; autologous use only
  • Minimum dose: 3 × 10⁶ CD34+ cells/kg body weight
  • Preceded by mobilisation and apheresis (collection target at least 20 × 10⁶ CD34+ cells/kg) and myeloablative conditioning
  • At least 2 × 10⁶ CD34+ cells/kg of unmodified back-up rescue cells are collected and cryopreserved
  • Mobilisation cycles separated by at least 14 days

Contraindications

  • Hypersensitivity to the active substance or any excipient
  • Contraindications of the mobilisation and conditioning medicines apply

Important warnings

  • Autologous use only; check patient identity against the labels and Lot information sheet
  • Hypersensitivity reactions including anaphylaxis (e.g. due to DMSO or dextran 40); observe during and after infusion
  • Risk of neutrophil engraftment failure; monitor ANC
  • Possible transmission of infectious agents
  • Traceability: keep product name, batch number and patient name for 30 years

Side effects

  • Safety profile generally consistent with busulfan conditioning and stem cell transplant
  • Very common attributed to the product: lymphopenia; common: thrombocytopenia, neutropenia, anaemia, headache, rash
  • Rare serious events: haemophagocytic lymphohistiocytosis, acute respiratory distress syndrome, delayed engraftment

Interactions

  • Discontinue hydroxyurea/hydroxycarbamide, voxelotor and crizanlizumab at least 8 weeks before mobilisation and conditioning
  • Interactions of the mobilisation and conditioning medicines must be considered

Pregnancy and breastfeeding

  • Negative pregnancy test before each mobilisation cycle and before conditioning; effective contraception from mobilisation through at least 6 months after conditioning
  • No data on excretion in human milk

Summary of the official product information, written with AI (Claude, Anthropic) on 2026-10-28. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.

Full official text →

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For patients

Package leaflet, verbatim

Package leaflet for patients →

Authorisation and brand names by country

Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.

EU – centrally authorised (all member states)

  • CasgevyVertex Pharmaceuticals (Ireland) Limited · EMEA/H/C/005763

Source: European Medicines Agency (EMA), product information

Brand names in 3 more European countries
Iceland
Casgevy
Liechtenstein
Casgevy
Norway
Casgevy

EMA – Article 57 product data (EU/EEA), 2026-10-28

Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.

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Sources

  1. European Medicines Agency. Casgevy – EPAR, Product Information (OF THE TEXT Detailed information on this medicinal product i). 2026-10-27. https://www.ema.europa.eu/en/documents/product-information/casgevy-epar-product-information_en.pdf

Editorial: Medelix editorial team · last updated 2026-10-27 · How these pages are made · Report an error

Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.