GSK receives European and CHMP approval for stem cell gene therapy

GlaxoSmithKline has been given encouraging support for a stem cell gene therapy that uses an individual patient's own cells to treat a very rare immunodeficiency disorder in children.

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, GSK's brand name for the medicine to treat the ADA-SCID disorder.

People with called ADA-SCID, which stands for severe combined immunodeficiency due to adenosine deaminase deficiency, lack virtually all immune protection from bacteria, viruses, and fungi, leaving them prone to repeated and persistent infections that can be very serious or life-threatening.

Glaxo said the CHMP opinion was an important step towards making Strimvelis available to the children living with this incredibly rare and fatal condition.

Going forward, the company hopes to apply the gene therapy platform technology across other diseases.

GSK had taken a gene therapy developed by Milan's San Raffaele Telethon Institute for Gene Therapy and optimised, standardised and characterised 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.

Martin Andrews, head of the rare disease unit at GSK, said: "This positive opinion is a major milestone in GSK's commitment to the development of innovative, transformative medicines.

"If approved, Strimvelis will become the first corrective ex-vivo gene therapy for children to achieve regulatory approval anywhere in the world. With our shared mission and complementary expertise we believe this collaboration will continue to deliver much needed new medicines for patients with rare genetic diseases."