GSK’s gene therapy Strimvelis gets European regulatory nod
UK drugmaker GlaxoSmithKline (LSE: GSK) said its gene therapy Strimvelis has secured backing from the European regulators.
The European Commission has granted marketing authorisation for Strimvelis to treat children with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), the company said in a joint statement with Fondazione Telethon and Ospedale San Raffaele.
Martin Andrews, head of the rare disease unit, GSK said: “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.”
The decision was based on data collected from 18 children treated with Strimvelis. A 100% survival rate at three years post-treatment with Strimvelis was observed for all children in the pivotal study, the company said in a statement.
ADA-SCID is a very rare disorder caused by a faulty gene inherited from both parents. This faulty gene stops the production of an essential protein called adenosine deaminase (ADA), which is required for the production of lymphocytes (a type of white blood cell). Children born with ADA-SCID do not develop a healthy immune system so cannot fight off everyday infections, which results in severe and life-threatening illness. Without prompt treatment, the disorder often proves fatal within the child’s first year of life. ADA-SCID is estimated to occur in about 15 patients per year in Europe.
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.
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