* 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 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
"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
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
"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
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)