Amicus Therapeutics (Nasdaq: FOLD) today announced that the Agency
of Medicines, Food and Medical Devices (ANMAT) in Argentina has
granted approval for the first-in-class, oral small molecule
pharmacological chaperone Galafold® (migalastat), as a monotherapy
for the treatment of people 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 approved
in Argentina.
Amicus is working with a local partner, Pint
Pharma, to complete the requirements to launch Galafold® in the
coming months. Argentina represents the first approval for
Galafold® in Latin America. Additional submissions for marketing
authorizations are under review or planned in this important
region.
“The approval of Galafold® in Argentina marks a significant step
for our oral precision medicine for Fabry disease in Latin
America,” stated John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics. “With more than 400 people known to
be living with Fabry disease in Argentina, we look forward to
collaborating with Pint Pharma, to bring our patient-dedicated
approach to the country. As many thousands more are estimated to be
living with Fabry in Latin America as a whole, Amicus and our
partners are committed to supporting people affected by the disease
throughout the region, and we believe a significant portion may
have amenable mutations that could benefit from treatment with
Galafold®.”
David Munoz, Chief Executive Officer of Pint
Pharma, stated, “The approval of Galafold® represents a major
milestone for patients living with Fabry disease in Argentina and
the region. We are honored to partner with Amicus and are committed
to bringing a patient focused vision by supporting early and
efficient access to innovative treatments.”
The Argentinian label for Galafold® includes over 350 GLA
mutations that have been identified and determined to be amenable
based on the Galafold Amenability Assay. It is estimated that
between 35% and 50% of people diagnosed with Fabry disease may have
an amenable GLA mutation.
Hernan Amartino, MD, Head of the Child Neurology
Department at Hospital Universitario Austral, Buenos Aires,
Argentina and Investigator in a migalastat clinical study, stated,
“The approval and pending availability of Galafold® is great news
for many Fabry patients in Argentina. Galafold® is the only
approved oral therapy for Fabry disease with a mechanism of action
designed to enable alpha-GAL A enzyme activity for patients with an
amenable mutation. Galafold® has demonstrated meaningful results in
previously untreated and treatment-experienced Fabry patients who
have amenable mutations, and at one capsule every other day, at the
same time of day, it is exciting for these patients to have a new
choice of treatment.”
Florencia Braga Menendez, Director of Patient
Affairs of Fundación Investigar, an organization committed to
access to healthcare in Argentina, added, “We welcome the approval
of Galafold® as the first oral precision medicine and first new
treatment option in more than a decade, for people living with
Fabry disease who have an amenable mutation in Argentina. We are
grateful to those who invest in researching and bringing hope to
families that await treatment for so many rare diseases. Even the
promise of a new treatment is powerful – it makes us feel less
invisible. Today is a big day for Fabry patients and for the
advancement of precision medicine in Argentina.”
In addition to Argentina, Galafold® is approved
in Australia, Canada, the European
Union, Israel, Japan, South
Korea, Switzerland and the U.S.
About Galafold® and Amenable
MutationsFabry 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. In patients with
an amenable mutation, Galafold® (migalastat) works by stabilizing
the body's own dysfunctional enzyme, so it can enter the
lysosome. Once inside the lysosome, Galafold® detaches from
the enzyme, allowing it to break down substrate. An amenable
mutation is one that results in an enzyme that can be stabilized by
Galafold®. Amenability is determined based on a proprietary in
vitro assay (Galafold Amenability Assay).
Healthcare providers in Argentina may access the
website www.galafoldamenabilitytable.com/ar to identify which
mutations are categorized as “amenable” or “not amenable” to
Galafold®. Amicus, through its partner Pint Pharma, expects to
submit additional updates to the website as additional GLA
mutations are identified and tested in the Galafold Amenability
Assay.
Important Argentine 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 non-amenable 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. The safety and efficacy of Galafold® in low birth
weight infants, neonates, nursing infants, infants and children
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: Headache and dizziness were the most common adverse
reactions reported at doses of migalastat of up to 1250 mg and 2000
mg, respectively, administrated in healthy subjects in the overseas
clinical studies.
- 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 Argentinian Galafold® 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 Argentina package insert
for Galafold®.
About Fabry DiseaseFabry
disease is an inherited lysosomal disorder caused by deficiency of
an enzyme called alpha-galactosidase A (alpha-Gal A), which results
from 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 heart, kidneys, and skin. Accumulation
of GL-3 and progressive deterioration of organ function is believed
to lead to the morbidity and mortality of Fabry disease. The
symptoms can be severe, differ from person to person, and begin at
an early age.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. With extraordinary patient focus, Amicus
Therapeutics is committed to advancing and expanding a robust
pipeline of cutting-edge, first- or best-in-class medicines for
rare metabolic diseases. For more information please visit the
company’s website at www.amicusrx.com, and follow on Twitter and
LinkedIn.
About Pint PharmaPint Pharma
International SA is a company registered under Swiss laws, having
its registered office at Route de Chenaux 9, 1091 Bourg-en-Levaux,
Switzerland, and is devoted to the development, registration, and
commercialization of specialty based treatments. Pint Pharma
benefits from leaders with extensive experience in the
pharmaceutical sector and who are based strategically throughout
Latin America and Europe. Pint Pharma has a long track record of
developing strong relationships with global pharmaceutical and
healthcare companies. Pint Pharma strives to be the first Pan-Latin
American provider of innovative and high value-added treatments
within Rare Diseases, Specialty Care, and Oncology.
Forward Looking StatementThis
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 Argentina. 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, 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: the potential that we may not be successful in
commercializing Galafold® in the Argentina, the potential that
Galafold® will not be reimbursed in Argentina, the potential that
we may not be able to manufacture or supply sufficient commercial
products; and the potential that we will need additional funding to
complete all of our commercialization and manufacturing activities.
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, 2018. 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:Amicus TherapeuticsMarco WinklerDirector,
Corporate Communicationsmwinkler@amicusrx.com(609) 662-2798
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