GlaxoSmithKline applies for EU approval of new rare disease therapy

GlaxoSmithKline, Fondazione Telethon and Ospedale San Raffaele have filed for European regulatory approval of a gene therapy for a rare disease.

A marketing application has been registered with the European Medicines Agency for GSK2696273, a new treatment for adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID).

The disease is characterised by an inability to produce lymphocytes that leaves patients with a severely deficient immune system. It is an extremely rare condition, with an estimated 14 children in Europe born with it each year.

GSK2696273 is intended for ADA-SCID patients for whom no suitable human leukocyte antigen-matched related stem cell donor is available. The marketing application is based on results from a successful trial involving 18 children

The first child was treated more than 13 years ago and all patients are still alive today.

Patrick Vallance, president of pharmaceutical research and development at GlaxoSmithKline, said: "We believe this marks a significant milestone, showing the potential of gene therapy as an important additional modality for tackling the underlying cause of serious diseases."