Strimvelis (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence)
OrphanATMPAdditional monitoring
- Region
- 🇪🇺 EU
- Active substance
- autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence
- Company
- Fondazione Telethon ETS
- Status
- Authorised
- Therapeutic area
- Severe Combined Immunodeficiency
- CHMP opinion
- Thu 31 Mar 2016
- EU approval
- Thu 26 May 2016
- Designations
- Orphan, ATMP, Additional monitoring
- Application
- EMEA/H/C/003854
- ATC
- L03
- Official record
- www.ema.europa.eu
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).
Regulatory milestones
No milestones recorded in the last year.
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.