CHMP recommends Strimvelis gene therapy to treat ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency)- GSK

GlaxoSmithKline has announced that 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 authorisation for 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 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 GSK 2696273 (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. Within the partnership GSK, working with the biotechnology company MolMed S.p.A, has applied its expertise in product development to optimise, standardise and characterise a manufacturing process that was previously only suitable for clinical trials into one that has been demonstrated to be robust and suitable for commercial supply.

Comment: Adenosine deaminase severe combined immunodeficiency syndrome- ADA-SCI. is an ultra rare disease in which patients cannot make lymphocytes (a type of white blood cell) and, as a result, have a severely deficient immune system. An estimated 14 children in Europe are born each year with the condition. A faulty gene inherited from both parents stops production of an essential protein called adenosine deaminase (ADA), which is particularly important for the formation of lymphocytes and a functioning immune system. Children born with ADA-SCID have an impaired ability to fight off everyday infections resulting in severe and life-threatening illness. They rarely survive beyond 1-2 years unless immune function is restored.