GlaxoSmithKline receives CHMP backing for EU approval of Strimvelis

GlaxoSmithKline has received a recommendation from Europe's Committee for Medicinal Products for Human Use (CHMP) for approval of Strimvelis.

The European Medicines Agency committee, in conjunction with the Committee for Advanced Therapies, has issued a positive opinion recommending marketing authorisation for this new therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).

ADA-SCID is a very rare genetic disorder that prevents the development of a health immune system. It is estimated to occur in approximately 15 patients per year in Europe, and often proves fatal within the first year of a child's life.

Strimvelis is a stem cell gene therapy created using the individual patient's own cells, which is intended to correct the root cause of the disease. If approved, it would be the first corrective ex-vivo gene therapy for children launched anywhere in the world.

Patrick Vallance, president of research and development at GlaxoSmithKline, said: "We welcome this opinion from the CHMP, which is an important step towards making Strimvelis available to the children living with this incredibly rare and fatal condition."

It is hoped that this gene therapy platform technology can be applied across other diseases in future.