Amicus Therapeutics Announces Chaperone-Advanced Replacement Therapy in Development for Mucopolysaccharidosis Type I (MPS I)

Amicus Therapeutics Announces Chaperone-Advanced Replacement Therapy in
Development for Mucopolysaccharidosis Type I (MPS I)

Grant Awarded to Support Preclinical Studies of Next-Generation Therapy

CRANBURY, N.J., June 25, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics
(Nasdaq:FOLD) today disclosed a preclinical Chaperone-Advanced Replacement
Therapy (CHART™) program for Mucopolysaccharidosis Type I (MPS I), a lysosomal
storage disease caused by missing or deficient alpha-L-iduronidase (IDUA)
enzyme. Amicus is developing a proprietary human recombinant IDUA (rhIDUA)
enzyme co-formulated with a novel pharmacological chaperone as a
next-generation therapy for MPS I.

The pharmacological chaperone is designed to improve tissue uptake and reduce
the immunogenicity of rhIDUA by stabilizing the enzyme in its properly folded
and active form. In addition, in support of its development of a proprietary
rhIDUA enzyme, Amicus has received a grant of up to approximately $250,000
from a private U.S.-based donor that provides medical research grants to find
better treatments and cures for rare genetic disorders, including lysosomal
storage diseases.

David J. Lockhart, Ph.D., Chief Scientific Officer of Amicus Therapeutics,
said, "This grant expands our preclinical work and supports our development of
a next-generation therapy for MPS I. Our goal is to engineer a proprietary,
potentially bio-better rhIDUA enzyme that may be further improved by the
addition of a pharmacological chaperone stabilizer. First-generation ERT for
MPS I has helped to improve certain manifestations of the disease, however,
rhIDUA is highly immunogenic. Directly co-formulating rhIDUA with a chaperone
has the potential to increase enzyme stability in the infusion bag and in
circulation, enhance uptake of active enzyme in disease-relevant tissues, and
potentially reduce immunogenicity."

In people with MPS I, missing or deficient IDUA leads to the accumulation of
complex carbohydrates that can affect physical abilities, organ and system
functioning, as well as mental and skeletal development. First-generation ERT
(laronidase) with rhIDUA has been shown to improve walking capacity and
pulmonary function in people with MPS I. However, most patients who receive
laronidase develop anti-rhIDUA antibodies, and the laronidase label carries a
black-box warning that cites life-threatening anaphylactic reactions in some
patients during infusion.^1 Amicus believes it may be able to improve upon the
properties of the rhIDUA enzyme itself, while also incorporating a
pharmacological chaperone stabilizer.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics,
stated, "Our MPS I program is an additional step forward for our CHART
platform technology to combine proprietary enzymes with novel pharmacological
chaperones across the lysosomal storage diseases. Importantly, the grant
funding supports our internal biologics capabilities at Amicus, which we are
leveraging alongside our expertise with chaperones to develop next-generation
therapies for people living with lysosomal storage diseases."

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the
forefront of therapies for rare and orphan diseases. The Company is developing
novel, first-in-class treatments for a broad range of human genetic diseases,
with a focus on delivering new benefits to individuals with lysosomal storage
diseases. Amicus' lead programs include the small molecule pharmacological
chaperones migalastat HCl as a monotherapy and in combination with enzyme
replacement therapy (ERT) for Fabry disease; and AT2220 (duvoglustat HCl) in
combination with ERT for Pompe disease.

About Chaperone-Advanced Replacement Therapy (CHART™)

The Chaperone-Advanced Replacement Therapy (CHART™) platform combines unique
pharmacological chaperones with enzyme replacement therapies (ERTs) for
lysosomal storage diseases (LSDs). In a chaperone-advanced replacement
therapy, a unique pharmacological chaperone is designed to bind to and
stabilize a specific therapeutic enzyme in its properly folded and active
form. This proposed CHART mechanism may allow for enhanced tissue uptake of
active enzyme, greater lysosomal activity, more reduction of substrate, and
lower immunogenicity compared to ERT alone. Improvements in enzyme stability
may also enable more convenient delivery of next-generation therapies. Amicus
is leveraging the CHART platform to develop pharmacological chaperones
co-administered with currently marketed ERTs as well as proprietary
next-generation therapies that consist of lysosomal enzymes co-formulated with
pharmacological chaperones.

About Mucopolysaccharidosis Type I (MPS I)

Mucopolysaccharidosis Type I (MPS I) is a severe, fatal, multi-system
lysosomal storage disease which can affect physical abilities, organ and
system functioning, as well as mental and skeletal development. It is caused
by a deficiency of the lysosomal enzyme alpha-L-iduronidase, which leads to
the accumulation of complex carbohydrates known as glycosaminoglycans (GAGs).

The incidence of MPS I is estimated to be at about 1 in 100,000 births. MPS I
is typically classified into three categories: 1) Hurler Syndrome, the most
severe form involving progressive developmental delay, physical problems, and
death within the first decade of life, 2) Hurler-Scheie Syndrome, an
intermediate form characterized by physical symptoms and normal or near-normal
cognitive ability, and 3) Scheie Syndrome, an attenuated form in which
individuals may have normal cognitive ability, average height, skeletal
deformities and an average life span.

^1Aldurazyme prescribing information, www.aldurazyme.com

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of
the Private Securities Litigation Reform Act of 1995 relating to clinical
development of Amicus' candidate drug products. Words such as, but not limited
to, "look forward to," "believe," "expect," "anticipate," "estimate,"
"intend," "plan," "targets," "likely," "will," "may", "potential", "would,"
"should" and "could," and similar expressions or words identify
forward-looking statements. Such forward-looking statements are based upon
current expectations that involve risks, changes in circumstances, assumptions
and uncertainties. The inclusion of forward-looking statements should not be
regarded as a representation by Amicus that any of its plans will be achieved.
Any or all of the forward-looking statements in this press release may turn
out to be wrong. They can be affected by inaccurate assumptions Amicus might
make or by known or unknown risks and uncertainties. For example, with respect
to statements regarding the potential goals, progress, timing and results of
preclinical studies and clinical trials, actual results may differ materially
from those set forth in this release due to the risks and uncertainties
inherent in the business of Amicus, including, without limitation: the
potential that results of clinical or pre-clinical studies indicate that the
product candidates are unsafe or ineffective; the potential that it may be
difficult to enroll patients in our clinical trials; the potential that
regulatory authorities may not grant or may delay approval for our product
candidates; the potential that preclinical and clinical studies could be
delayed because we identify serious side effects or other safety issues; the
potential that we will need additional funding to complete all of our studies
and, our dependence on third parties in the conduct of our clinical studies.
Further, the results of earlier preclinical studies and/or clinical trials may
not be predictive of future results. In addition, all forward looking
statements are subject to other risks detailed in our Annual Report on Form
10-K for the year ended December 31, 2012. You are cautioned not to place
undue reliance on these forward-looking statements, which speak only as of the
date hereof. All forward-looking statements are qualified in their entirety by
this cautionary statement, and Amicus undertakes no obligation to revise or
update this news release to reflect events or circumstances after the date
hereof. This caution is made under the safe harbor provisions of Section 21E
of the Private Securities Litigation Reform Act of 1995.

FOLD–G

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