27 May 2016
GlaxoSmithKline (GSK), Ospedale San Raffaele (OSR), and Fondazione Telethon (Telethon) have announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy for the treatment of patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency). A child born with ADA-SCID does not have an immune system that is healthy and fully-functioning. Consequently, this system is not able to fight off everyday infections. It has been estimated that approximately 15 children per year are affected by ADA-SCID in Europe.
“Today’s approval is the result of many years’ work with our collaborators in Milan and is the next step towards bringing life-changing treatment to patients with ADA-SCID and their families. This is the start of a new chapter in the treatment of rare genetic diseases and we hope that this therapeutic approach could also be used to help patients with other rare diseases in the future,” said Martin Andrews, Head of the Rare Disease Unit, GSK.
As commented by Nicola Bedin, CEO of Ospedale San Raffaele: “This great achievement would not have been possible without the effective collaboration between OSR, Telethon and GSK, which has brought together years of scientific research, first-class medical practice and expertise in product development. Going forward we hope to build on our shared mission to develop and deliver more much-needed new medicines to patients with rare diseases.”