Amicus Therapeutics (Nasdaq:FOLD) announced today that Japan's
Ministry of Health, Labour and Welfare (MHLW) has approved the oral
small molecule pharmacological chaperone Galafold® capsules 123mg
(migalastat) for treatment of patients aged 16 years and older with
a confirmed diagnosis of Fabry disease (alpha-galactosidase A
deficiency) and who have an amenable mutation. Galafold is the
first and only oral precision medicine for Fabry disease in Japan.
Amicus will now proceed with pricing and reimbursement processes,
and anticipates launching Galafold in Japan in the coming months
once those processes have concluded.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc., stated, “The Japanese
approval for Galafold is a major step forward for more than 800
people currently known to be living with Fabry disease in Japan. We
believe a significant portion of these Fabry patients have amenable
mutations that are suitable for treatment with this differentiated
precision oral therapy. I would like to highlight the tremendous
collaboration among our Amicus employees, Japanese regulators, and
the Fabry community, in particular those physicians and patients
who participated in the clinical studies of Galafold and their
families who made this approval possible. Japan is very important
to our patient-focused vision to provide Galafold to Fabry patients
with amenable mutations throughout the world as soon as possible.
And now that Amicus has established a strong presence in Japan, we
hope that the upcoming Galafold launch will be the first of many
future opportunities to deliver new medicines for people living
with rare metabolic diseases in Japan.”
Fabry disease is a rare genetic disease and
potentially life-threatening condition caused by the accumulation
of disease substrate (globotriaosylceramide, GL-3) in the lysosome
due to a dysfunctional or deficient enzyme. Galafold works by
stabilizing the body's own dysfunctional enzyme, so it can clear
the accumulated disease substrate in patients who have amenable
mutations. An amenable mutation is one that is responsive to
therapy with migalastat based on a proprietary in vitro assay
(Galafold Amenability Assay).
Prof. Toya Ohashi, Jikei University, stated, “As
a principal investigator in the Galafold pivotal studies with
extensive experience treating Fabry disease, it can be said that
significant unmet need remains. Galafold has a unique mechanism of
action that has demonstrated compelling results in naïve and
treatment-experienced Fabry patients who have amenable mutations.
This differentiated treatment option is good news for the many
Fabry patients in Japan who have an amenable mutation.”
Yoshiyuki Suzuki, M.D., Ph.D., Former Professor,
International University of Health and Welfare Graduate School,
said, “I am pleased with Amicus’ leadership in advancing Galafold
to approval. Galafold is part of a chaperone technology platform
that originated here in Japan, and represents an important step
forward within the field of personalized medicine in Japan.”
Mr. Hisao Harada, Chair of the Japan Fabry
Disease Patients and Family Association (JFA) commented, “The
approval of Galafold is great news for Fabry patients in Japan as
it provides the first new Fabry treatment option in more than a
decade. We welcome this oral precision medicine and look forward to
it being available to a number of Fabry patients in Japan who have
amenable mutations."
An estimated 850 people in Japan are living with
Fabry disease. Japan represented approximately 13% of the $1.3B
global Fabry ERT sales generated in Japan in 2017.1 Galafold was
approved by the MHLW under the priority review scheme allowed for
an Orphan Drug. The approval was based on clinical data from
completed clinical studies of Galafold, including 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 a Phase 1 study that evaluated the
pharmacokinetics of migalastat in Japanese volunteers.
Galafold is currently reimbursed in 18
countries, on a commercial basis or through expanded access
programs (EAPs). The European Commission granted full approval for
Galafold in May 2016 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 and who have an amenable
mutation. Outside the EU, marketing applications have been approved
in six markets, including Australia, Canada, Israel, South Korea,
Switzerland, and now Japan. Approvals of Galafold are currently
pending in the U.S. and Taiwan.
About Galafold and Amenable
MutationsGalafold® capsules 123 mg (migalastat) is a
first-in-class chaperone therapy approved in Japan 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) was used to classify more than 1,000 known GLA
mutations as “amenable” or “not amenable” to treatment with
Galafold. Amicus estimates that 35%-50% of Fabry patients globally
may have amenable genetic mutations, and amenability rates within
this range vary by geography.
Healthcare providers in the Japan 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 website as additional GLA mutations are identified and
tested in the Galafold Amenability Assay.
Important Japanese Safety
InformationTreatment with GALAFOLD should be initiated and
supervised by specialists experienced in the diagnosis and
treatment of Fabry disease. GALAFOLD is not indicated for use in
patients with a nonamenable mutation.
- The efficacy and the safety of concomitant use with enzyme
replacement therapy has not been established. 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.
- Migalastat exposure is affected by food, therefore it should
not be taken within 2 hours before and after food.
- Patients should be observed carefully, and caution should be
taken in the administration in the elderly population.
- 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, and consider the treatment
only in case that the benefit from migalastat is judged to exceed
the risk during pregnancy. Nursing mothers should be instructed not
to breast-feed if they are taking migalastat or to discontinue
migalastat if they do breast-feed
- Contraindications to GALAFOLD include hypersensitivity to the
active substance or to any of the excipients listed in the
PRESCRIBING INFORMATION.
- Patients should be monitored based on their course including
renal and cardiac function and clinical laboratory test during
migalastat treatment. In case no effect is observed in the
migalastat treatment, changing treatment should be considered.
- 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 Japan package insert.
- Call your doctor for medical advice about side effects.
For further important safety information for Galafold, including
dosage and administration, precautions, drug interactions and
adverse drug reactions, please see the Japan package insert for
Galafold available at
http://www.pmda.go.jp/PmdaSearch/iyakuSearch/.
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 TherapeuticsAmicus
Therapeutics (Nasdaq:FOLD) is a global, patient-centric
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. The cornerstone of the Amicus portfolio is
migalastat, an oral precision medicine for people living with Fabry
disease who have amenable genetic mutations. Migalastat is
currently approved under the trade name Galafold™ in the European
Union, with additional approvals granted and pending in several
geographies. The lead biologics program in the Amicus pipeline is
ATB200/AT2221, a novel, late-stage, potential best-in-class
treatment paradigm for Pompe disease. The Company is committed to
advancing and expanding a robust pipeline of cutting-edge, first-
or best-in-class medicines for rare metabolic diseases.
1Company filings and Amicus estimates
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to approval and commercialization of Galafold in Japan.
The inclusion of forward-looking statements should not be regarded
as a representation by us that any of our plans will be achieved.
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
pricing regulatory authorities, actual results may differ
materially from those set forth in this release due to the risks
and uncertainties inherent in our business, and the potential that
we may not be successful in commercializing Galafold in Japan. 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, 2017. 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, IRCVice President, Investor Relations
& Corporate Communicationsspellegrino@amicusrx.com (609)
662-5044
Media:Pure CommunicationsJennifer Paganelli
jpaganelli@purecommunications.com (347) 658-8290
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