| Clinical data | |
|---|---|
| Trade names | Strimvelis |
| License data | |
| Routes of administration | Intravenous |
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| Legal status | |
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| Identifiers | |
| DrugBank | |
Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).[1]
ADA-SCID is a rare inherited condition in which there is a change (mutation) in the gene needed to make an enzyme called adenosine deaminase (ADA).[1] As a result, people lack the ADA enzyme.[1] Because ADA is essential for maintaining healthy lymphocytes (white blood cells that fight off infections), the immune system of people with ADA-SCID does not work properly and without effective treatment they rarely survive more than two years.[1]
Strimvelis is the first ex vivo autologous gene therapy approved by the European Medicines Agency (EMA).[2]
- ^ a b c d e "Strimvelis EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 8 October 2023. Retrieved 11 June 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ "Pipeline". Orchard Therapeutics. 2 July 2021. Archived from the original on 15 June 2021. Retrieved 5 July 2021.
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