AMT's Marketing Authorisation Application for Glybera(R) Progressing on Schedule

By Amsterdam Molecular Therapeutics B.v, PRNE
Tuesday, August 17, 2010

AMSTERDAM, The Netherlands, August 18, 2010 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in
the field of human gene therapy, today announced that the Marketing
Authorisation Application (MAA) for Glybera(R) remains on schedule following
meetings with the European Medicine Agency (EMA) concerning the Day 120 List
of Questions. The company is confident that Glybera(R), a gene therapy
product for lipoprotein lipase deficiency (LPLD), remains on track for a
regulatory decision by mid-2011.

AMT has had two meetings with the Committee for Advanced
Therapy Medicinal Products (CAT) at the EMA for clarification about the Day
120 questions. These meetings have enabled AMT to finalise its strategy for
responding to these questions in a timely and effective manner.

The outcome of the meetings suggests that AMT will not be
required to conduct more clinical trials with additional new patients to be
treated at this time. The responses to the questions will be based in part on
additional data and analyses from patients previously treated with
Glybera(R). This will include new data available from the last clinical trial
(CT-AMT-011-02) and its one year extension.

"We have developed a clear response strategy which, if
executed with no unforeseen adverse events or delays, should allow us to
remain on track for an EMA decision in the middle of 2011," noted Jörn Aldag,
CEO of Amsterdam Molecular Therapeutics. "The route to registration for an
innovative product such as Glybera(R) is not only very important for AMT, but
it also gives hope to thousands of patients suffering from rare diseases.
Gene therapy carries the promise to be able to cure a range of diseases
thought to be caused by a single gene. AMT will exert every effort to succeed
and reach the market with this unique product."

About the Disease

LPLD is a seriously debilitating orphan disease for which no
treatment exists today. The disease is caused by mutations in the LPL gene,
resulting in highly decreased or absent activity of LPL protein in patients.
This protein is needed in order to break down large fat-carrying particles
that circulate in the blood after each meal. When such particles, called
chylomicrons, accumulate in the blood, they may obstruct small blood vessels.
This can result not only in potentially lethal pancreatitis, but also in
difficult-to-treat diabetes, and is associated with significant morbidity and

About Amsterdam Molecular Therapeutics

AMT is a leader in the development of human gene based
therapies. Using adeno-associated viral (AAV) derived 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. This proprietary platform can be applied to a large number of rare
(orphan) diseases that are caused by one faulty gene. Currently, AMT has a
product pipeline with several AAV-based gene therapy products in LPLD,
Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and,
Parkinson's Disease at different stages of research or development. AMT was
founded in 1998 and is based in Amsterdam.


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. EMA, 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.

For further enquiries: Jörn Aldag CEO, Tel +31(0)20-566-7394, Mob +31(0)6-8195-3060, j.aldag at

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