Amicus Therapeutics Launches Galafold™ (Migalastat) for Treatment of Fabry Disease in Spain
January 17 2018 - 4:01PM
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
at the forefront of rare and orphan diseases, has commenced the
commercial launch of the precision medicine Galafold in Spain
following final pricing and reimbursement decisions. Galafold is
now reimbursed in Spain 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 commercial launch of Galafold in Spain adds
to the tremendous momentum in expanding our launch throughout the
EU, where we have now secured reimbursement in all five countries
that have the largest Fabry populations within the EU,” stated John
F. Crowley, Chairman and Chief Executive Officer of Amicus
Therapeutics, Inc. “We believe that our pricing and reimbursement
success throughout the EU, in addition to our global progress in
securing additional approvals and completing new regulatory
submissions, are a testament to the significant value of migalastat
as the first oral precision medicine for people living with Fabry
disease who have an amenable mutation.”
Fabry disease is a genetic disease which causes
deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A),
which is the result of mutations in the GLA gene. As a precision
medicine, Galafold is designed to restore alpha-Gal A activity in
patients who have amenable mutations (an estimated 35% to 50% of
the Fabry population).
Dr. Roser Torra, Chief of Inherited Renal
Diseases Unit. Nephrology Department, Fundación Puigvert, Barcelona
stated, “There are unmet medical needs in Fabry disease, and oral
Galafold represents a therapeutic alternative for Fabry patients
with amenable mutations. Galafold is a new paradigm in precision
medicine due to its innovative mechanism of action.”
Jordi Cruz, Director of the Association of
Mucopolysaccharidosis and Related Syndromes, MPS Spain, said, “It
is great news each time a new treatment for rare diseases becomes
available. The approval of this new treatment also represents a
differentiated innovation in the treatment of this type of
lysosomal storage disease. Patients with Fabry disease living in
Spain who have an amenable mutation are fortunate to now have
access to this oral precision medicine.”
The European Commission granted the first
approval for Galafold 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,
migalastat is approved in Switzerland, Israel, Australia, Canada
and South Korea, with regulatory submissions under review in
additional geographies.
About Galafold™ and Amenable
MutationsGalafold™ (migalastat) is a first-in-class
chaperone therapy approved in the EU 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. The
current EU label includes 313 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 label as additional GLA mutations are identified and tested
in the Galafold Amenability Assay.
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 nonamenable 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, 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 future value driver of 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.
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995.
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 commercialization of Galafold, 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 Spain, Europe and other geographies or
our other product candidates if and when approved. 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. 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 Paganelli jpaganelli@purecommunications.com
(347) 658-8290
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