AMSTERDAM, January 30, 2012 /PRNewswire/ –

Amsterdam Molecular Therapeutics (Euronext:
AMT –
News), a leader in the field of human gene therapy, was
informed on Friday January 27, 2012 after business hours that
the European Commission's Standing Committee of the European
Parliament discussed on Monday January 22, 2012 the
implementation decision not to grant marketing authorization
for Glybera (alipogene tiparvovec) as recommended by the
Committee for Human Medicinal Products (CHMP). After the
discussion no clear position in favor or against granting a
marketing authorization for Glybera was reached. Instead the
Standing Committee considered it necessary to request
additional information to the CHMP in the European Medicines
Agency (EMA). A formal vote by the Standing Committee will be
made on review of the additional information. It is currently
unclear when a final decision will be reached.

Glybera is a gene therapy for patients with the genetic
disorder lipoprotein lipase deficiency (LPLD). Currently no
alternative treatment is available for patients deficient of
LPL, who are suffering from recurrent pancreatitis.

On October 23, 2011, AMT received a non-approvable opinion from
the CHMP (following a re-examination of the marketing approval
dossier originally rejected in June 2011) despite a
recommendation to approve Glybera by the Committee For Advanced
Therapies (CAT). The CAT is an expert body implemented by the
European Commission to prepare and advise the CHMP on decisions
regarding advanced therapeutics, in particular gene and cell
therapies.

“We are very encouraged by the outcome of the discussion by the
European Parliament's Standing Committee to request further
information from the CHMP. The evaluation of (ultra-) orphan
drugs and advanced therapies is novel territory. The ongoing
debate shows that the authorities are determined to find the
best process of dealing with such innovative treatments and
technologies. We will constructively work with the European
Commission and the EMA to find the best process going forward,
not only for Glybera but also for further advanced therapies on
their way to patients,” said Jörn Aldag, CEO of AMT. “While
gaining clarity for the process involving Glybera, we will
continue to implement our revised business strategy revealed
following the last CHMP decision. This involves moving forward
in partnering discussions for several of our gene therapy
products, including hemophilia B and GDNF.”

AMT's current business plan, organizational structure and
financing have been developed on the basis of the current CHMP
opinion for Glybera's non-approval. It involved a reduction of
its workforce by about 50%. In the event that Glybera could be
commercialized in Europe, the Company will evaluate how the
opportunity arising from passing this important milestone could
be realized most efficiently.

About Glybera®

AMT has developed Glybera as a treatment for patients with the
genetic disorder lipoprotein lipase deficiency. LPLD is an
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. Excess chylomicrons
result in recurrent and severe acute inflammation of the
pancreas, called pancreatitis, the most debilitating and
life-threatening clinical complication of LPLD. Glybera has
orphan drug status in the EU and US.

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene
based therapies. In addition to Glybera, AMT has a product
pipeline of several gene therapy products in development for
hemophilia B, Duchenne muscular dystrophy, acute intermittent
porphyria, Parkinson's disease and SanfilippoB. 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 probably the world's first stable
and scalable AAV manufacturing platform. This proprietary
platform can be applied to a large number of rare (orphan)
diseases caused by one faulty gene and allows AMT to pursue its
strategy of focusing on this sector of the industry.  AMT
was founded in 1998 and is based in Amsterdam. Further
information can be found at

http://www.amtbiopharma.com.

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 AMT
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. AMT expressly disclaims
any intent or obligation to update any forward-looking
statements herein except as required by law. 

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Amsterdam Molecular Therapeutics Announces European Commission Delays Decision on Glybera® Marketing Authorisation and …