GSK and partners submit EU marketing application for ultra-rare disease

UK pharma major GlaxoSmithKline (LSE: GSK), Fondazione Telethon and Ospedale San Raffaele have submitted a marketing application to the European Medicines Agency for a gene therapy to treat patients with a rare disease.

The therapy, GSK2696273, has been submitted for approval to treat the rare disease adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID), for patients where no suitable human leukocyte antigen-matched related stem cell donor is available.

ADA-SCID is an ultra-rare disease in which patients cannot make lymphocytes so have a severely deficient immune system, and 14 children per year are born in Europe with the condition. They have an impaired ability to fight off everyday infections, resulting in severe and life-threatening illnesses, and rarely survive beyond 1-2 years of age unless immune function is restored. Most require a bone marrow transplant from a matched donor.