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EMA recommends first new gene therapy

Published on 24/07/12 at 10:01am
uniQure's Glybera
uniQure's Glybera

Glybera is now on the cusp of becoming the first gene therapy treatment to be approved in the West.

The CHMP is recommending that Glybera, which is developed by Amsterdam-based uniQure, be used in patients with severe or multiple pancreatitis attacks due to lipoprotein lipase deficiency.

“We expect final approval from the European Commission within three months after the CHMP decision,” says Jörn Aldag, chief executive of uniQure. 

“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,” he added.

If approved the drug will be the first gene therapy to reach a Western market - there is already an approved gene therapy in China.

Glybera could have peak annual sales between €50 million ($61 million) and €300 million, depending on how the firm prices its drug.

The EMA’s decision comes after 40 years of research into gene therapies, many of which have been unsuccessful.

But the uniQure’s success could also open the doors for more gene therapy treatments, and revitalise firms such as Sangamo BioSciences and Neurologix, both of which are developing their own gene therapies.

Glybera has not had an easy road - it originally sent its drug for European review in 2009, but was rejected by the CHMP in 2011.

But in an unusual move, the European Commission asked the Agency to look again at the drug by restricting the patient population to those with severe or multiple pancreatitis attacks.

By looking at just this particular group, the EMA’s committees felt they could now recommend the treatment.

The treatment targets lipoprotein lipase deficiency, or LPLD, a rare fat-processing disorder that spurs severe or multiple pancreatitis attacks in about one or two people among every million in the population.

The therapy needs only be administered once to be effective.

As part of the approval, treatment with Glybera will be offered through centres of excellence with expertise in treating LPLD.

It will also be administered by specially trained doctors to ensure its ongoing safety, due to it being such a new therapy with uncertainty surrounding its long-term effects.

The company said it is now preparing to apply for regulatory approval in the US, Canada, and other markets.

Ben Adams

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