EMA Authorised: Strimvelis (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) — Fondazione Telethon ETS

View source ↗

Summary

EMA authorised Strimvelis for the treatment of severe combined immunodeficiency due to ADA deficiency (ADA-SCID); the marketing authorization holder is Fondazio…

Upgrade for the full analysis, importance score, and drug timeline.

Diseases / indications

• Combined Immunodeficiency

Drugs mentioned

• Strimvelis

Snippet

Status: Authorised. Therapeutic area: Severe Combined Immunodeficiency. MAH: Fondazione Telethon ETS. Decision date: 04/07/2025. Orphan medicine