EMA Starts Formal Review of Glybera(R) Dossier

By Amsterdam Molecular Therapeutics B.v, PRNE
Sunday, January 24, 2010

AMSTERDAM, The Netherlands, January 25 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in
the field of human gene therapy, has reached another important milestone in
the official marketing authorisation process for its lead product Glybera(R),
AMT's proprietary product for lipoprotein lipase deficiency (LPLD). The
submission of the Glybera(R) Marketing Authorisation Application (MAA),
announced earlier, has cleared the validation stage with The European
Medicines Agency (EMA, formerly known as EMEA). The EMA will now commence its
formal review of Glybera(R).

AMT has concluded two clinical studies for LPLD, in Europe and
Canada, and long term follow-up from both of these is ongoing, as is a third
clinical study in Canada. In these three studies Glybera(R) has shown a
sizeable decrease in the incidence of pancreatitis, or acute inflammation of
the pancreas, the most debilitating complication of LPLD. In addition, these
studies indicate that Glybera(R) has an excellent safety profile.

"The acceptance of the Glybera(R) dossier by EMA is a
significant step towards marketing approval for Glybera(R). Moreover, it
demonstrates AMT's development capability. A future approval of the MAA for
Glybera would fully validate our gene therapy approach and our
adeno-associated viral (AAV) vector delivery platform. We believe this step
offers hope to many patients, as gene therapy may become the therapeutic
approach of choice for inherited disorders" said Jörn Aldag, Chief Executive
Officer of AMT.

The EMA formal review will be conducted via the centralised
procedure, which is the standard route for all advanced therapies. During
2010, AMT expects to provide further updates on the results from its
follow-up and ongoing studies, in accordance with reporting regulations.

About the Disease

LPLD is a seriously debilitating, and potentially lethal,
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 pancreatitis, but
also in difficult-to-treat diabetes, and is associated with significant
morbidity and mortality.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in
the development of human gene based therapies. Using AAV 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 safe and efficacious proprietary platform offers a unique
manufacturing capability which 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 LPL
Deficiency, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson's
Disease at different stages of research or development.

For information

AMT will be presenting at the BioCEO & Investor Conference,
Waldorf-Astoria, New York City, at 9:30 am (EST) on Tuesday, February 9,
2010
.

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.

For information: Jörn Aldag, Chief Executive Officer, Tel +31(0)20-566-7394, j.aldag at amtbiopharma.com .

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