This is a summary of the European public assessment report (EPAR) for Strimvelis. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Strimvelis. For practical information about using Strimvelis, patients should read the package leaflet or contact their doctor or pharmacist.
Therapeutic Indication
### Therapeutic indication 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).
Therapeutic Area (MeSH)
N/AATC Code
L03
ATC Item
免疫促进药
Pharmacotherapeutic Group
Immunostimulants
Active Substance (Summary)
INN / Common Names
N/A| Substance | CAS | Monograph |
|---|---|---|
| autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells | N/A | autologous cd 34+ enriched cell fraction that contains cd 34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ada) cdna sequence from human haematopoietic stem/progenitor (cd 34+) cells |
EMA Name
Strimvelis
Medicine Name
Strimvelis
Aliases
N/ANo risk management plan link.
