UPDATE 1-AMT gene therapy rejected again in EU, shares dive

Fri Oct 21, 2011 2:14pm BST

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* EU drugs agency maintains view Glybera not approvable

* CEO says "surprised", will continue to pursue gene therapy

* AMT shares plunge 55 percent (Adds company comment, latest shares, background)

By Ben Hirschler and Aaron Gray-Block

LONDON/AMSTERDAM, Oct 21 (Reuters) - Amsterdam Molecular Therapeutics' novel gene therapy drug Glybera has again been rejected by European regulators, underscoring the difficulty of getting gene-based treatments to market.

Shares in the Dutch biotech company plunged 55 percent on the news on Friday.

Glybera is designed for patients with a rare genetic disorder called lipoprotein lipase deficiency (LPLD), whom AMT Chief Executive Jorn Aldag said would now be left without a viable treatment.

"The decision from the CHMP is disappointing particularly because a treatment will now not be available to European patients who suffer from LPLD for whom Glybera is the only therapeutic option," he said.

LPLD can cause acute pancreatitis, diabetes and death.

The London-based European Medicines Agency, which first rebuffed Glybera in June, reiterated its view that the drug was not approvable because there was insufficient evidence that its benefits outweighed its potential risks.

The decision, however, was a close call. The agency's Committee for Advanced Therapies had expressed a view that Glybera could be approved, subject to further post-marketing studies. But the Committee for Medicinal Products for Human Use, which has the final say, disagreed.

"There is strong endorsement for our technology platform and for Glybera to be approved under exceptional circumstances, but it has not happened. I am just surprised," Aldag told Reuters.

"This company stands out and has a validated programme and therefore we will continue to develop gene therapy," he said, adding there was interest from pharma companies for AMT's hemophilia programme.

Aldag said AMT has funding to last into the first quarter of next year, but the company has a strong shareholder base and investors are willing to look at funding options.

AMT will provide an update on its gene therapy pipeline on Oct. 25.

"I think for people who understand gene therapy, it is evident there is tremendous value here," Aldag said.

More than 20 years after the first experiments with the ground-breaking technology of fixing faulty genes, scientists and drug companies are still struggling to apply gene therapy in everyday medicine.

Last year, Britain's Ark Therapeutics pulled its bid to sell brain cancer drug Cerepro in Europe following a series of setbacks. Cerepro was the first gene-based medicine to be considered for European approval. (Editing by David Cowell)

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