Strimvelis
What Strimvelis is used for
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available (see section 4.2 and section 4.4).
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.
Key facts
- Brand
- Strimvelis
- Active substance
- autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence
- Disease area
- Severe Combined Immunodeficiency
- Medicine group
- Immunostimulants
- ATC code
- L03
- Marketing authorisation holder
- Fondazione Telethon ETS
- Authorised in the EU since
- 2016
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 Strimvelis used for?
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available (see section 4.2 and section 4.4).
What is the active substance in Strimvelis?
Strimvelis contains autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence.
Who makes Strimvelis?
The EU marketing authorisation holder is Fondazione Telethon ETS.
Is Strimvelis an orphan medicine?
Yes. Strimvelis is designated an orphan medicine in the EU, for a rare disease (Severe Combined Immunodeficiency).
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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