Amicus Therapeutics (Nasdaq:FOLD) announced that the U.S. Food and
Drug Administration (FDA) has granted Fast Track designation for
the oral precision medicine migalastat for the treatment of
patients with Fabry disease with amenable mutations. The FDA’s Fast
Track program is designed to expedite the development and review of
drugs and biologics with the potential to treat serious or
life-threatening conditions and with nonclinical or clinical data
that demonstrate the potential to address unmet medical needs.1
Fabry disease is a progressive, inherited
lysosomal storage disorder caused by an enzyme deficiency. The
disease causes accumulation of specific lipids in tissues including
the central nervous system, heart, kidneys, and skin. This abnormal
accumulation can lead to debilitating consequences including pain,
kidney failure, heart disease, and stroke.
“This Fast Track designation recognizes that
Fabry is a serious disease and that migalastat has the potential to
address the significant needs faced by this patient community,”
stated John F. Crowley, Chairman and Chief Executive Officer of
Amicus Therapeutics, Inc. “Migalastat is an oral precision medicine
that may offer an important new treatment choice in the U.S. for
people with Fabry who have not had a new treatment option in almost
fifteen years. As we execute our international launch and continue
pursuing global regulatory approvals for migalastat, it is our
vision to bring this important treatment to even more people in
more geographies who may benefit. We look forward to submitting our
NDA and collaborating with the U.S. FDA throughout the regulatory
process.”
The Company plans to submit a New Drug
Application (NDA) for migalastat in the fourth quarter of 2017.
Drugs with Fast Track designation may qualify for accelerated
approval and priority review to expedite the FDA review process, if
relevant criteria are met. This designation also provides frequent
communication with the FDA throughout the review process.
Migalastat works by stabilizing the body's own
dysfunctional enzyme, so it can clear the accumulated disease
substrate in patients who have amenable mutations (an estimated 35%
to 50% of Fabry patients globally). An estimated 3,000 people
in the U.S. are currently diagnosed with Fabry disease, more than
any other country.
The European Commission (EC) granted full
approval for migalastat, under the trade name Galafold™, as a first
line therapy for long-term treatment of adults and adolescents aged
16 years and older with a confirmed diagnosis of Fabry disease
(alpha-galactosidase A deficiency) and who have an amenable
mutation. The EC approval was based on clinical data from two
Phase 3 pivotal studies in both treatment-naïve (Study 011, or
FACETS) and enzyme replacement therapy (ERT) switch patients (Study
012, or ATTRACT), as well as ongoing long-term extension
studies.
Outside the EU, migalastat is approved in
Switzerland, Israel, Australia and Canada, with regulatory
submissions under review in Japan and additional geographies.
About Fast Track
Designation1Fast Track designation is intended to
facilitate development and expedite review of drugs to treat
serious and life-threatening conditions. A drug that is intended to
treat a serious condition with nonclinical or clinical data that
demonstrate the potential to address unmet medical need may qualify
for Fast Track designation. When Fast Track designation is
requested later in development, available clinical data should
demonstrate the potential to address an unmet medical need. There
are opportunities for frequent interactions with the review team
for a fast track product. In addition, such a product could be
eligible for priority review if supported by clinical data at the
time of BLA, NDA, or efficacy supplement submission.
About Galafold™ and Amenable
MutationsGalafold™ (migalastat) is a first-in-class
chaperone therapy approved in the European Union as a monotherapy
for Fabry disease in patients with amenable mutations. Galafold
works by stabilizing the body’s own dysfunctional enzyme, so it can
clear the accumulation of disease substrate in patients who have
amenable mutations. A proprietary in vitro assay (Galafold
Amenability Assay) has been used to classify more than 1,000 known
GLA mutations as “amenable” or “not amenable” to treatment with
Galafold. The EU label includes 331 GLA mutations that have been
identified and determined to be amenable based on the Galafold
Amenability Assay, which represent between 35% and 50% of the
currently diagnosed Fabry population.
Healthcare providers in the EU may access the
website www.Galafoldamenabilitytable.com to quickly and accurately
identify which mutations are categorized as “amenable” or “not
amenable” to Galafold. Amicus expects to submit additional updates
to the EU label as additional GLA mutations are identified and
tested in the Galafold Amenability Assay.
EU Important Safety
InformationTreatment with Galafold should be initiated and
supervised by specialists experienced in the diagnosis and
treatment of Fabry disease. Galafold is not recommended for use in
patients with a non-amenable mutation.
- Galafold is not intended for concomitant use with enzyme
replacement therapy.
- Galafold is not recommended for use in patients with Fabry
disease who have severe renal impairment (<30 mL/min/1.73 m2).
The safety and efficacy of Galafold in children 0–15 years of age
have not yet been established.
- No dosage adjustments are required in patients with hepatic
impairment or in the elderly population.
- There is very limited experience with the use of this medicine
in pregnant women. If you are pregnant, think you may be pregnant,
or are planning to have a baby, do not take this medicine until you
have checked with your doctor, pharmacist, or nurse.
- While taking Galafold, effective birth control should be used.
It is not known whether Galafold is excreted in human milk.
- Contraindications to Galafold include hypersensitivity to the
active substance or to any of the excipients listed in the
PRESCRIBING INFORMATION.
- It is advised to periodically monitor renal function,
echocardiographic parameters and biochemical markers (every 6
months) in patients initiated on Galafold or switched to
Galafold.
- OVERDOSE: General medical care is recommended in the case of
Galafold overdose.
- The most common adverse reaction reported was headache, which
was experienced by approximately 10% of patients who received
Galafold. For a complete list of adverse reactions, please review
the SUMMARY OF PRODUCT CHARACTERISTICS.
- Call your doctor for medical advice about side effects.
For further important safety information for
Galafold, including posology and method of administration, special
warnings, drug interactions and adverse drug reactions, please see
the European SmPC for Galafold available from the EMA website at
www.ema.europa.eu.
About Fabry DiseaseFabry
disease is an inherited lysosomal storage disorder caused by
deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A),
which is the result of mutations in the GLA gene. The primary
biological function of alpha-Gal A is to degrade specific lipids in
lysosomes, including globotriaosylceramide (referred to here as
GL-3 and also known as Gb3). Lipids that can be degraded by the
action of alpha-Gal A are called "substrates" of the enzyme.
Reduced or absent levels of alpha-Gal A activity lead to the
accumulation of GL-3 in the affected tissues, including the central
nervous system, heart, kidneys, and skin. Progressive accumulation
of GL-3 is believed to lead to the morbidity and mortality of Fabry
disease, including pain, kidney failure, heart disease, and stroke.
The symptoms can be severe, differ from patient to patient, and
begin at an early age. All Fabry disease is progressive and may
lead to organ damage regardless of the time of symptom onset.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company
at the forefront of therapies for rare and orphan diseases. The
Company has a robust pipeline of advanced therapies for a broad
range of human genetic diseases. Amicus’ lead programs in
development include the small molecule pharmacological chaperone
migalastat as a monotherapy for Fabry disease, as well as novel
enzyme replacement therapy (ERT) and biologic products for Fabry
disease, Pompe disease, and other rare and devastating
diseases.
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to the clinical development, regulatory approval pathway,
and prospects and timing of regulatory submission and approval of
our product candidates for the treatment of Fabry disease. Any
express or implied statements contained in this press release that
are not statements of historical fact, including interpretation of
guidance given by the U.S. FDA may be deemed forward-looking
statements. The inclusion of forward-looking statements should not
be regarded as a representation by us that any of our plans will be
achieved in a timely manner or at all. Any or all of the
forward-looking statements in this press release may turn out to be
wrong and can be affected by inaccurate assumptions we might make
or by known or unknown risks and uncertainties. For example, with
respect to statements regarding the goals, progress, timing, and
outcomes of discussions with regulatory authorities, actual results
may differ materially from those set forth in this release due to
the risks and uncertainties inherent in our business, including,
without limitation, changes in FDA guidance for regulatory
approval, risks regarding the FDA’s interpretation of our clinical
trial results, including the risk that results from completed
clinical trials that supported approval by regulators in other
jurisdictions will not be sufficient for U.S. FDA purposes, the
risk that the FDA will require additional studies or data, the risk
that the timing of an NDA will be delayed or not be accepted by the
FDA, the potential that regulatory authorities, including the FDA,
EMA, and PMDA, may not grant or may delay approval for our product
candidate and the potential that we may not be successful in
commercializing our product candidates for Fabry disease in Europe
or any other country in which approval is ultimately obtained, if
any. 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, 2016 and the Quarterly Report for the quarter
ended June 30, 2017. The FDA guidance described in this release was
given as of a specific date and the FDA could change its position
on the clinical end points or other standards for review and/or
approval. 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 we undertake no obligation to revise
or update this news release to reflect events or circumstances
after the date hereof.
1http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf
CONTACTS:
Investors/Media:Amicus
TherapeuticsSara Pellegrino, IRCSenior Director, Investor
Relationsspellegrino@amicusrx.com (609) 662-5044
Media:W2O GroupJennifer Paganelli
jpaganelli@purecommunications.com (347) 658-8290
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