| April 01, 2016, 10:26:00 AM EDT By RTT News |
(RTTNews.com) - GlaxoSmithKline (GSK, GSK.L) announced that the Committee for Medicinal Products for Human Use or CHMP of the European Medicines Agency or EMA, in conjunction with the Committee for Advanced Therapies or CAT, has issued a positive opinion recommending marketing authorisation for Strimvelis to treat patients with a very rare disease called ADA-SCID or severe combined immunodeficiency due to adenosine deaminase deficiency.
The medicine is a stem cell gene therapy created for an individual patient from their own cells which is intended to correct the root cause of the disease. If approved by the European Commission, the medicine - currently known as GSK2696273 (autologous CD34+ cells transduced to express ADA) - will be commercialised under the brand name Strimvelis, for the treatment of patients with ADA-SCID for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
The gene therapy for the treatment of ADA-SCID was originally developed by Ospedale San Raffaele (OSR) and Fondazione Telethon (Telethon), through their joint San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and was taken forward by GSK through a strategic collaboration formed in 2010 between GSK, OSR and Telethon.
Read more: http://www.nasdaq.com/article/gsk-gets-positive-chmp-opinion-for-strimvelis-to-treat-rare-disease-adascid-20160401-00467#ixzz44f5JneSs