Last updated on April 19, 2014 at 18:42 EDT

First Gene Therapy in Western World Receives Positive Opinion in Europe from CHMP

July 20, 2012

AMSTERDAM, The Netherlands, July 20, 2012 /PRNewswire/ –

        - First gene therapy in the Western world to reach important regulatory
          approval milestone, culminating 40 years of research
        - First therapy for LPL deficient patients, a severe disease with no alternative
        - Validates uniQure's unique AAV-based gene therapy platform, consisting of a
          modular, plug-and-play vector system and unrivaled GMP manufacturing capabilities on a
          commercial scale
        - Heralds new phase in uniQure's development, including potential revenues from
          sales and partnerships
        - Technology platform can now be leveraged to find solutions for many more
          severe genetic and other disorders

uniQure announced today that the European Medicines Agency’s Committee for Medicinal
Products for Human Use (CHMP) has issued a positive opinion that recommends marketing
authorization of Glybera(R) (alipogene tiparvovec) as a treatment for lipoprotein lipase
deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease.
Patients with the disease are unable to handle fat particles in their blood plasma, which
leads to recurring severe abdominal pain and pancreatitis.

The European Commission (EC) generally follows the recommendations of the CHMP. “We
expect final approval from the EC within 3 months after the CHMP decision,” says Jorn
Aldag, CEO of uniQure. “After today’s positive recommendation, Glybera is poised to become
the first in a class of gene therapy products approved in Europe to treat orphan diseases,
rare conditions with a very high unmet medical need.” Marketing authorization covers all
27 European Union member states.

Mr. Aldag continued: “Patients with LPLD are afraid of eating a normal meal because it
can lead to acute and extremely painful inflammation of the pancreas, often resulting in a
visit to intensive care. Now, for the first time, a treatment exists for these patients
that not only reduces this risk of getting severely sick, but also has a multi-year
beneficial effect after just a single injection. The positive recommendation from the CHMP
for Glybera therefore represents a major breakthrough for both LPLD patients and for
medicine as a whole. Restoring the body’s natural ability to break down fat particles in
the blood in order to prevent pancreatitis and excruciating abdominal pain suffered by
patients, is what gene therapy is all about: curing disease at the genetic level.”

“At uniQure we are developing treatments for a number of other rare diseases as well,
such as acute intermittent porphyria and Sanfilippo B. But the potential of gene therapy
stretches far beyond rare diseases. As shown recently in a publication in the New England
Journal of Medicine (N Engl J Med 2011; 365:2357-2365, December 22, 2011), hemophilia
patients treated with our proprietary gene are showing a sustained clinical effect over
several years, which has allowed prophylaxis treatment to be stopped. In addition, we are
advancing programs in degenerative diseases such as Parkinson’s. We believe that just like
antibodies, gene therapy will one day be a mainstay in clinical practice,” Mr Aldag added.

As part of the approval, treatment with Glybera will be offered through dedicated
centers of excellence with expertise in treating LPLD and by specially trained doctors to
ensure ongoing safety of this novel treatment paradigm. uniQure has also committed to
building a patient registry for continued understanding of this devastating,
under-researched disease. The Company is now preparing to apply for regulatory approval in
the US, Canada, and other markets.

Glybera has been tested in three interventional clinical studies conducted in the
Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three
clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data
from these clinical trials indicate that a single dose administration of Glybera resulted
in a long-term biological activity of the LPL protein.

About Glybera(R)

uniQure has developed Glybera as a therapy 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 complication of LPLD. Glybera has
orphan drug designation in the EU and US. LPL Deficiency affects 1-2 persons per million.
For further information on LPLD visit http://www.lpldeficiency.com.

About uniQure

uniQure is a world leader in the development of human gene based therapies. uniQure
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 uniQure to pursue its strategy of
focusing on this sector of the industry. uniQure’s largest shareholders are Forbion
Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital
firms in the Netherlands. Further information can be found at http://www.uniqure.com.

SOURCE uniQure B.V.

Source: PR Newswire