June 24, 2011 / 8:30 AM / 8 years ago

UPDATE 1-Gene therapy faces setback as EU rebuffs AMT drug

* AMT shares slump 50 pct on blow to lead product Glybera

* CEO still hopes for EU green light with more data

* Drug would be first gene therapy approved in EU (Adds CEO interview, latest shares, background)

By Ben Hirschler

LONDON, June 24 (Reuters) - A novel gene therapy drug from Amsterdam Molecular Therapeutics has been rejected by European regulators, underscoring the difficulty of getting gene-based treatments to market.

Shares in the Dutch biotech company slumped 50 percent on the news on Friday, although AMT said it still believed its product Glybera could win a positive opinion if it generated additional data from existing patients.

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.

AMT Chief Executive Jorn Aldag told Reuters he was optimistic the Dutch company would not face the same fate.

“Obviously is a disappointment for investors if you are expecting an approval and it is not confirmed — but from our perspective this is a delay and not a definitive answer,” he said in a telephone interview.

Glybera is designed for patients with a rare genetic disorder called lipoprotein lipase deficiency (LPLD). The London-based European Medicines Agency, however, decided that trial results to date provided insufficient proof of its clinical benefit.

Aldag said he aimed to answer this by examining more data from existing patients who have already been given the drug, which will take until the end of this year but will not involve any extra expense.

Because LPLD is a very rare disease, only a small number of patients have tested the medicine and AMT expects to look at results from between five and 10 patients in the coming months to generate the extra data.

LPLD can cause acute pancreatitis. The disease may also lead to diabetes and death.

In the past, AMT has said it aimed to strike a partnership deal this year for Glybera or another product in its pipeline, and Aldag said he believed such a tie-up was still possible.

That could bring a welcome cash injection for the loss-making group, although Aldag said AMT had enough money to continue operations until early next year.

“We are strong enough financially to see us through this,” he said. (Reporting by Ben Hirschler; Editing by Erica Billingham)

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