EMA Advisory Committee Recommends GSK’s Gene Therapy for Rare DiseaseBy
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), in conjunction with the Committee for Advanced Therapies (CAT), has issued a positive opinion recommending marketing authorization for GlaxoSmithKline’s (GSK) Strimvelis to treat patients with a very rare disease called ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency). The medicine is a stem-cell gene therapy created for an individual patient from his/her 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 commercialzsed 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. Within the partnership GSK, working with the biotechnology company MolMed S.p.A, has applied its expertise in product development to optimize, standardize,and characterize a manufacturing process that was previously only suitable for clinical trials into one that has been demonstrated to be suitable for commercial supply.
ADA-SCID is a very rare disorder caused by a faulty gene inherited from both parents. This faulty gene stops the production of an essential protein called adenosine deaminase (ADA), which is required for the production of lymphocytes (a type of white blood cell). Children born with ADA-SCID do not develop a healthy immune system so cannot fight off everyday infections, which results in severe and life-threatening illness. Without prompt treatment, the disorder often proves fatal within the child's first year of life. ADA-SCID is estimated to occur in approximately 15 patients per year in Europe.