European regulator clears uniQure's Glybera as first gene therapy product

The European Commission approved uniQure's Glybera (alipogene tiparvovec) for patients with lipoprotein lipase deficiency (LPLD) suffering from recurring acute pancreatitis, the company reported Friday, making the product the first gene therapy approved by regulators in the Western world. CEO Jörn Aldag remarked that the decision "marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need."

The approval had been expected after the European Medicines Agency's Committee for Medicinal Products for Human Use issued a positive opinion on Glybera in July. The therapy is specifically indicated for adults with familial LPLD suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. The company noted that the diagnosis of LPLD has to be confirmed by genetic testing and Glybera will be administered at dedicated centres by specialist doctors. Clinical trials in 27 patients with LPLD showed that a single dose of uniQure's therapy resulted in long-term biological activity of the LPL protein.

According to uniQure, Glybera will be launched in Europe in the second half of next year, and the company is currently working with governments on potential pricing strategies. Aldag suggested that some countries preferred the idea of a one-off payment at the time of treatment, while others were interested in an annuity system, which would likely involve charging approximately 250 000 euros ($321 700) a year for five years. Aldag said that the price of Glybera was due to the fact it "provides higher benefit to patients than the classical protein replacement strategy." UniQure indicated that it is also preparing to apply for regulatory approval of Glybera in the US, Canada and other markets.

Shenzhen SiBiono GeneTech's product for head and neck cancer was approved in China in 2003, making it the first ever gene therapy authorised.

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