GSK, Fondazione Telethon and Ospedale San Raffaele have announced that the EMA has approved Strimvelis for a super rare immunodeficiency disease in Children.
Following 20 years of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Italy, this is the news patients have been waiting for.
Strimvelis is the first ex vivo stem cell gene therapy to treat patients with the very rare type of Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase deficiency (ADA-SCID).
ADA-SCID affects an estimated 15 children per year in Europe and following today’s approval, patients with the condition who are referred for treatment will be able to receive the gene therapy at Ospedale San Raffaele university hospital in Milan.
But this requires frequent injections and is not as effective as it could be,