GSK becomes first big pharma to seek gene therapy approval
This article was originally published in SRA
Britain's biggest drug maker, GlaxoSmithKline, is seeking regulatory approval in Europe to use gene transfer technology in children born with ADA-SCID and has further programs using the approach in two other rare diseases, metachromatic leukodystrophy and Wiskott-Aldrich syndrome, with both currently in clinical trials1.
GSK has become the first big pharma to file for marketing approval of a gene therapy by submitting GSK2696273 for ADA-severe combined immune deficiency in Europe. GSK's five-year-old rare diseases unit plans follow-up indications using the same therapy aimed at fixing faulty genes, but a similar regulatory filing in the US isn't imminent, the company says.
The investigational gene therapy has not been approved for use anywhere in the world but is being given accelerated review by the European Medicines Agency. Its submission for approval in Europe, announced May 5, is the result of a five-year collaboration between GSK, the Italian biomedical charity Fondazione Telethon, and the San Raffaele Telethon Institute for Gene Therapy, a research center for stem cell gene therapy based in Milan, Italy.
Martin Andrews, senior vice president of global rare diseases at GSK said it could take up to 1-1.5 years for the EMA to give its verdict on GSK2696273's filing. "This is breakthrough medicine which the EMA must evaluate, so it’s hard to say how long it will take. Realistically I take the assumption of between 12 to 18 months. The minimum would be 6 months for an option, but it’s more likely that it will be a normal review timeframe. For any NME for the EMA to consider, the review time is on average 15 months - so it could be anywhere between 12 to 15 months."
A longer version of this article was published in The Pink Sheet Daily (see GSK Is First Big Pharma To Seek Gene Therapy Approval, Plans More, 7 May 2015)
References
1. GSK press release, 5 May 2015, www.gsk.com/en-gb/media/press-releases/2015/gsk-fondazione-telethon-and-ospedale-san-raffaele-announce-eu-regulatory-submission-for-gene-therapy-to-treat-rare-disease-ada-scid/