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Last updated on April 17, 2014 at 21:23 EDT

AMT Receives EMEA Orphan Drug Designation for Duchenne Muscular Dystrophy

October 13, 2009

AMSTERDAM, October 13 /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-080 for the treatment of
Duchenne muscular dystrophy.

Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitles
AMT to ten year market exclusivity in Europe following marketing approval for
AMT-080 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 Duchenne muscular dystrophy,” said Jorn Aldag, Chief Executive
Officer of AMT. “This designation is an important next step in the
development of a treatment for this progressive and devastating disease.”

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe disease characterized by
progressive muscle degeneration. It affects young children, almost
exclusively boys, and leads to paralysis and death in young adulthood. The
disease is caused by mutations in the dystrophin gene, thereby blocking the
production of functional dystrophin protein, an important structural
component within muscle tissue. Currently there is no treatment to prevent
the fatal outcome of this disease. DMD affects one in 3,500 males, making it
the most prevalent of muscular dystrophies.

AMT is developing a gene therapy product for Duchenne muscluar dystrophy
based on technology that results in “skipping” of the defective portion of
the dystrophin gene resulting in the formation of functional protein.
Positive long-term health effects of this approach have been demonstrated in
animals.

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), especially diseases 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