Last updated on April 21, 2014 at 1:20 EDT

Amsterdam Molecular Therapeutics Receives Request for Further Information From CHMP on Glybera®

February 21, 2012

AMSTERDAM, The Netherlands, February 21, 2012 /PRNewswire/ –

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene
therapy, announced today that it has received a request to provide additional information
to the Committee for Human Medicinal Products (CHMP) on Glybera(R) (alipogene tiparvovec)
by March 8, 2012. The CHMP request follows the announcement on January 30, 2012 that the
European Commission Standing Committee was requesting additional information from the
CHMP. The CHMP has indicated that a final decision on Glybera could be made by May 2012.

Glybera is a gene therapy for patients with the genetic disorder LPLD, a very rare
inherited condition that is associated with increased levels of chylomicrons. These
particles carry certain types of fat in the blood, which because they are not removed from
the body can cause recurrent bouts of pancreatitis. There is no treatment currently
available for these patients.

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.

AMT’s proposed acquisition of assets by uniQure BV announced on February 17, 2012, was
developed on the basis that Glybera is not approvable. This transaction will proceed as
planned. In the event that Glybera could be commercialized in Europe, uniQure will
evaluate how achieving this important milestone could be realized most efficiently.

About Glybera(R)

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. AMT has a
product pipeline of gene therapy products in development for hemophilia B, 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.

About uniQure

uniQure BV is a private company created specifically for the transaction with AMT. It
is funded by Forbion Capital Partners, an existing investor in AMT. uniQure will act as
the new holding company for the gene therapy business currently carried out by AMT.

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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indirectly, in or into the United States or to US persons. This announcement is not a
prospectus and does not contain or constitute an offer for sale or the solicitation of an
offer to purchase securities in the United States or any other jurisdiction.

The securities mentioned herein have not been and will not be registered under the
U.S. Securities Act of 1933, as amended (the “Securities Act”), or under the securities
laws of any state or other jurisdiction of the United States and may not be offered, sold,
resold, taken up, exercised, renounced, transferred or delivered, directly or indirectly,
within the United States except pursuant to an applicable exemption from, or in a
transaction not subject to, the registration requirements of the Securities Act. No public
offering of the securities mentioned herein is being made in the United States or any
other jurisdiction.

This announcement does not constitute or forms a part of any offer or solicitation to
purchase or subscribe for securities in any jurisdiction or jurisdictions in which such
offers or sales are unlawful prior to registration or qualification under the securities
laws of any such jurisdiction.

The materials contained herein have not been submitted to or reviewed by the US
Securities and Exchange Commission (the “SEC”) or any state securities commission, and
neither the SEC nor any such state securities commission has (a) approved or disapproved,
(b) passed upon the merits or fairness of, or (c) passed upon the adequacy or accuracy of
the disclosure of any materials contained herein. Any representation to the contrary is a
criminal offence in the United States.

SOURCE Amsterdam Molecular Therapeutics (AMT) B.V

Source: PR Newswire