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EC gives green light to UniQure's £1m gene therapy

Glybera approved to treat rare, inherited condition lipoprotein lipase deficiency

Gene therapy graphic - UniQure

Gene therapy graphic (UniQure)

After decades of research and development, a gene therapy has finally been approved for marketing in the EU and looks set to be launched next year.

Dutch company UniQure has been granted approval for Glybera (alipogene tiparvovec), a treatment for patients with the inherited disease lipoprotein lipase deficiency (LPLD).

Glybera is the first gene therapy to be approved in the western world, although a treatment in this category has been available in China since 2004, in the form of Shenzhen SiBiono Gene Technologies' Gendicine for head and neck cancer.

LPLD – also called familial hyperchylomicronemia – is extremely rare, affecting no more than 1-2 people per million, and is characterised by an inability to metabolise fat particles in the blood, which causes pancreatitis and, in some cases, can be life-threatening.

Glybera's approval means that LPLD patients have access to a treatment for the first time, according to vascular medicine specialist Professor John Kastelein of the University of Amsterdam in the Netherlands. He said it "will have a dramatic impact on the lives of these patients".

Eyebrows are already being raised about the price tag of the gene therapy, however, with a one-time course costing around £1m ($1.6m), although pricing and reimbursement details will be the subject of negotiations between UniQure and EU governments.

Patients would be treated with the gene therapy, which is administered by intramuscular injection, at specialist centres and by doctors trained to deliver the treatment. UniQure said it will also build a patient registry to help improve the understanding of LPLD.

The approval is a validation not only of a gene therapy approach to disease, but also UniQure's adeno-associated virus (AAV) technology that is used to carry Glybera's genetic payload, said the company's chief executive Jörn Aldag.

The green light "offers strong support for our other advanced development programs, which focus on acute intermittent porphyria, Sanfilippo B, haemophilia B and Parkinson's disease", he said.

Gene therapies, which alter a patient's genetic code, have had a challenging time navigating through regulatory channels, and Glybera was no exception. It was turned down twice by the EC's Committee for Medicinal Products for Human Use (CHMP) before finally getting a positive opinion in June 2012.

5th November 2012

From: Sales

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