First western-world 'gene therapy' drug receives approval in Europe

Quote from UniQure press release

UniQure announced today that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances.

gene therapy for genetic TMAU ?

Gene Therapy has been seen as a Holy Grail cure for metabolic disorders, especially disorders where a single enzyme is at fault. However since gene therapy stated to be researched between 10-20 years ago, not one drug has received approval in the western world by the appropriate regulatory bodies. Finally, a gene therapy drug has received approval in Europe, to become the first approved gene therapy drug in the western world.

The drug, Glybera, is for the rare metabolic disorder lipoprotein lipase deficiency, a disorder that currently has no alternative treatment.

The drug has been developed by UniQure in the Netherlands. The website says gene therapy is particularly suitable for single enzyme disorders, which would be the case for FMO3 deficiency and probably any other metabolic malodor disorders that have not been recorded yet.As well as already progressing in some trials for other rare metabolic disorders, they also expect it can treat other disorders such as Parkinson's disease and hearing loss, which UniQure are already investigating.

Below are some quotes regarding Glybera from the Uniqure press release

"First gene therapy in the Western world to reach important regulatory approval milestone, culminating 40 years of research"

"Validates uniQure’s unique AAV-based gene therapy platform, consisting of a modular, plug-and-play vector system and unrivaled GMP manufacturing capabilities on a commercial scale"

"After today’s positive recommendation, Glybera is poised to become the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need.” Marketing authorization covers all 27 European Union member states."

"Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection. The positive recommendation from the CHMP for Glybera therefore represents a major breakthrough for both LPLD patients and for medicine as a whole. Restoring the body's natural ability to break down fat particles in the blood in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level."

"At uniQure we are developing treatments for a number of other rare diseases as well, such as acute intermittent porphyria and Sanfilippo B. But the potential of gene therapy stretches far beyond rare diseases. As shown recently in a publication in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365, December 22, 2011), hemophilia patients treated with our proprietary gene are showing a sustained clinical effect over several years, which has allowed prophylaxis treatment to be stopped. In addition, we are advancing programs in degenerative diseases such as Parkinson’s. We believe that just like antibodies, gene therapy will one day be a mainstay in clinical practice"

"The Company is now preparing to apply for regulatory approval in the US, Canada, and other markets."

"Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein."

Press Release on UniQure website

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Links
NY Times article on Glybera being first western-world gene therapy drug to get approval

Glybera article in UK newspaper The Guardian

Gene therapy nears approval in Europe, BBC News Health

Gene therapy and stem cells unite, BBC News Health