Amicus Therapeutics (Nasdaq:FOLD) submitted a new drug application
(NDA) to the U.S. Food and Drug Administration (FDA) to request
approval of the oral precision medicine migalastat HCl
(“migalastat”) for the treatment of patients 16 years and older
with Fabry disease who have amenable mutations. The NDA submission
is based on existing clinical data, including reduction in
disease-causing substrate (GL-3), as well as the totality of data
from two Phase 3 pivotal studies in treatment-naïve (Study 011, or
FACETS) and enzyme replacement therapy (ERT) switch patients (Study
012, or ATTRACT), as well as other completed clinical studies.
Migalastat previously received both Orphan Drug
Designation and Fast Track designation from the U.S. FDA. The FDA
has a 60-day filing review period to determine whether the NDA is
complete and acceptable for filing, after which the Agency will
notify the Company. Amicus plans to communicate following written
receipt of the Agency’s decision.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc., stated, “Today marks the very
first Amicus submission of a new drug application to the U.S. FDA.
This important milestone is the culmination of a strong
collaboration and commitment among the patients, physicians and
Amicus employees who have spent more than a decade to advance
migalastat. On the heels of our initial launch success for
migalastat in Europe, our goal is to further expand access for more
Fabry patients with amenable mutations in the U.S., Japan and other
global geographies. We look forward to working with the FDA
during the review process and to a potential U.S. approval of
migalastat in 2018.”
An estimated 3,000 people in the U.S. are
currently diagnosed with Fabry disease, more than any other
country. Fabry disease is a progressive, 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 disease causes accumulation of
specific lipids, primarily GL-3, in tissues including the heart,
kidneys, central nervous system, and skin. This abnormal
accumulation can lead to debilitating consequences including pain,
kidney failure, heart disease, and stroke.
Migalastat works by stabilizing the body's own
dysfunctional alpha-Gal A enzyme, so it can clear the accumulated
disease substrate in patients who have amenable mutations (an
estimated 35% to 50% of Fabry patients globally). 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 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 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 for
filing 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 September 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.
CONTACTS:
Investors/Media:Amicus
TherapeuticsSara Pellegrino, IRCSenior Director, Investor
Relationsspellegrino@amicusrx.com (609) 662-5044
Media:Pure CommunicationsJennifer
Paganellijpaganelli@purecommunications.com (347) 658-8290
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