GSK receives positive CHMP opinion in Europe for Strimvelis, the first gene therapy to treat very rare disease, ADA-SCID

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.

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, 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.

Patrick Vallance, President, R&D, GSK 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. Going forward, we hope to apply this gene therapy platform technology across other diseases, enabling many more patients to benefit from this innovative treatment approach.”

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.”

Until the EU commission decision, Strimvelis will remain an investigational gene therapy which is not approved for use anywhere in the world.