AMT Receives EMEA Orphan Drug Designation for Duchenne Muscular Dystrophy

Thursday, October 15, 2009 General News
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AMSTERDAM, October 13 Amsterdam MolecularTherapeutics (Euronext: AMT), a leader in the field of human gene therapy,announced today that the European Medicines Agency has granted Orphan DrugDesignation to AMT's gene therapy product AMT-080 for the treatment ofDuchenne muscular dystrophy.

Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitlesAMT to ten year market exclusivity in Europe following marketing approval forAMT-080 if this product candidate is the first new drug with a major medicalbenefit receiving marketing approval for the European Union. The designationalso provides for special benefits, including research support, eligibilityfor protocol assistance and possible exemptions or reductions in certainregulatory fees during development or at the time of application formarketing approval.

"We are proud to have received this Orphan Drug Designation for thetreatment of Duchenne muscular dystrophy," said Jorn Aldag, Chief ExecutiveOfficer of AMT. "This designation is an important next step in thedevelopment of a treatment for this progressive and devastating disease."

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe disease characterized byprogressive muscle degeneration. It affects young children, almostexclusively boys, and leads to paralysis and death in young adulthood. Thedisease is caused by mutations in the dystrophin gene, thereby blocking theproduction of functional dystrophin protein, an important structuralcomponent within muscle tissue. Currently there is no treatment to preventthe fatal outcome of this disease. DMD affects one in 3,500 males, making itthe most prevalent of muscular dystrophies.

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

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumventmany if not all of the obstacles that have prevented gene therapy frombecoming a mainstay of clinical medicine. Using adeno-associated viral (AAV)vectors as the delivery vehicle of choice for therapeutic genes, the companyhas been able to design and validate what is probably the first stable andscalable AAV production platform. As such, AMT's proprietary platform holdstremendous promise for thousands of rare (orphan), especially diseases thatare caused by one faulty gene. Currently, AMT has a product pipeline withnine 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 futureoperations, prospects and financial condition. Words such as "strategy,""expects," "plans," "anticipates," "believes," "will," "continues,""estimates," "intends," "projects," "goals," "targets" and other words ofsimilar meaning are intended to identify such forward-looking statements.Such statements are based on the current expectations of the management ofAmsterdam Molecular Therapeutics only. Undue reliance should not be placed onthese statements because, by their nature, they are subject to known andunknown risks and can be affected by factors that are beyond the control ofAMT. Actual results could differ materially from current expectations due toa number of factors and uncertainties affecting AMT's business, including,but not limited to, the timely commencement and success of AMT's clinicaltrials and research endeavors, delays in receiving U.S. Food and DrugAdministration or other regulatory approvals (i.e. EMEA, Health Canada),market acceptance of AMT's products, effectiveness of AMT's marketing andsales efforts, development of competing therapies and/or technologies, theterms of any future strategic alliances, the need for additional capital, theinability to obtain, or meet, conditions imposed for required governmentaland regulatory approvals and consents. AMT expressly disclaims any intent orobligation to update these forward-looking statements except as required bylaw. For a more detailed description of the risk factors and uncertaintiesaffecting AMT, refer to the prospectus of AMT's initial public offering onJune 20, 2007, and AMT's public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V

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