Strimvelis

Strimvelis
Clinical data
Trade namesStrimvelis
Routes of
administration		Intravenous
ATC code
  • None
Legal status
Legal status
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]

  1. ^ 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 copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  2. ^ "Pipeline". Orchard Therapeutics. 2 July 2021. Archived from the original on 15 June 2021. Retrieved 5 July 2021.