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AMT Receives EMEA Orphan Drug Designation for Acute Intermittent Porphyria

May 28, 2009

AMSTERDAM, May 28 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (Euronext: AMT), a leader in the field of human gene therapy,
announced today that the European Medicines Agency has granted Orphan Drug
Designation to AMT’s gene therapy product AMT-021 for the treatment of acute
intermittent porphyria (AIP).

Orphan Drug Designation for AIP entitles AMT to ten year market
exclusivity in Europe following marketing approval for AMT-021 if this
product candidate is the first new drug with a major medical benefit
receiving marketing approval for the European Union. The designation also
provides for special benefits, including research support, eligibility for
protocol assistance and possible exemptions or reductions in certain
regulatory fees during development or at the time of application for
marketing approval.

“We are proud to have received this Orphan Drug Designation for the
treatment of acute intermittent porphyria,” said Sander van Deventer, Chief
Executive Officer of AMT. “This designation is an important step in the
development of a treatment for this seriously debilitating and potentially
lethal disease.”

About Acute Intermittent Porphyria

Acute intermittent porphyria is a rare genetic disease in which mutations
in the PBGD gene result in insufficient activity of a protein necessary for
the synthesis of heme. This leads to an accumulation of toxic intermediate
metabolites resulting in a wide variety of problems including acute, severe
abdominal pains, psychiatric, neurologic illnesses, and muscular weakness.
Long-term consequences may be irreversible nerve damage, liver cancer and
kidney failure. Acute porphyric attacks can be life-threatening.

AIP affects 1 per 10.000 people in the European Union.

Currently available therapies do not prevent the symptoms and
consequences of acute porphyric attacks. AMT has demonstrated that its
product, AMT-021, results in normalization of the PBGD protein in an animal
model of AIP. In this model, the therapy completely prevented the occurrence
of attacks and significantly ameliorated the neuropathy that develops in
untreated mice. AMT-021 is intended to provide long-term normalization of the
PBGD protein in order to prevent acute porphyric attacks and their
complications.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumvent
many if not all of the obstacles that have prevented gene therapy from
becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV)
vectors as the delivery vehicle of choice for therapeutic genes, the company
has been able to design and validate what is probably the first stable and
scalable AAV production platform. As such, AMT’s proprietary platform holds
tremendous promise for thousands of rare (orphan) diseases, especially those
that are caused by one faulty gene. Currently, AMT has a product pipeline
with nine products at different stages of development.

Certain statements in this press release are “forward-looking statements”
including those that refer to management’s plans and expectations for future
operations, prospects and financial condition. Words such as “strategy,”
“expects,” “plans,” “anticipates,” “believes,” “will,” “continues,”
“estimates,” “intends,” “projects,” “goals,” “targets” and other words of
similar meaning are intended to identify such forward-looking statements.
Such statements are based on the current expectations of the management of
Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on
these statements because, by their nature, they are subject to known and
unknown risks and can be affected by factors that are beyond the control of
AMT. Actual results could differ materially from current expectations due to
a number of factors and uncertainties affecting AMT’s business, including,
but not limited to, the timely commencement and success of AMT’s clinical
trials and research endeavors, delays in receiving U.S. Food and Drug
Administration or other regulatory approvals (i.e. EMEA, Health Canada),
market acceptance of AMT’s products, effectiveness of AMT’s marketing and
sales efforts, development of competing therapies and/or technologies, the
terms of any future strategic alliances, the need for additional capital, the
inability to obtain, or meet, conditions imposed for required governmental
and regulatory approvals and consents. AMT expressly disclaims any intent or
obligation to update these forward-looking statements except as required by
law. For a more detailed description of the risk factors and uncertainties
affecting AMT, refer to the prospectus of AMT’s initial public offering on
June 20, 2007, and AMT’s public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V


Source: newswire



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