CHMP recommends first ever gene therapy for Western approval
24 July 2012 16:48 in Scientific Developments/Breakthroughs
The lipoprotein lipase deficiency (LPL) treatment Glybera could become the first gene therapy to be approved for use in the Western world, following a recommendation from the Committee for Medicinal Products for Human Use (CHMP).
Developed by uniQure, the compound has been endorsed for use under exceptional circumstances and would also be the first treatment ever to be approved for this extremely rare hereditary disease.
The condition can cause a severe and painful inflammation of the pancreas following a normal meal, but Glybera is able to restore the body's natural ability to break down fat particles in the blood in order to prevent pancreatitis.
Full European approval of the drug is expected within the next three months, bringing 40 years of research to a successful conclusion.
Jorn Aldag, chief executive officer of uniQure, said: "We believe that just like antibodies, gene therapy will one day be a mainstay in clinical practice."
Glybera is one of many gene therapies being developed by uniQure, which allied last month with the University of California, San Francisco to create a genetic treatment for Parkinson's disease.
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