Amicus Therapeutics Announces Approval for Galafold™ (Migalastat) for Treatment of Fabry Disease in Canada
September 14 2017 - 4:01PM
Amicus Therapeutics (Nasdaq:FOLD) announced that Health Canada has
approved the oral precision medicine Galafold for long-term
treatment of adults with a confirmed diagnosis of Fabry disease
[deficiency of alpha-galactosidase (alpha-Gal A)] and who have an
alpha-Gal A mutation determined to be amenable by an in vitro
assay. Following the Health Canada approval, Amicus expects to make
Galafold available to Canadian patients in the coming weeks.
"The approval of Galafold represents a
significant step forward for the Canadian Fabry community,” stated
John F. Crowley, Chairman and Chief Executive Officer of Amicus
Therapeutics, Inc. “We look forward to launching Galafold in Canada
to further advance our mission of expanding global access to
Galafold for people living with Fabry disease who have amenable
mutations.”
Health Canada approved Galafold 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 an ongoing
long-term extension study and overall body of evidence. Fabry 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
accumulation of disease substrate in patients who have amenable
mutations. An amenable mutation is one that is responsive to
therapy with Galafold based on a proprietary in vitro assay
(Galafold Amenability Assay).
"Following more than a decade of experience
treating Fabry patients with Galafold in the clinical setting, I am
pleased that Fabry patients in Canada who have amenable mutations
will soon have access to this important and differentiated
medicine,” said Dr. Daniel Bichet, Full Professor and Section Head,
Renal Function & Transport Physiology, University of Montreal,
and Principal Investigator for Canada in the Galafold clinical
studies. “Galafold has a unique mechanism of action that is rooted
in the underlying genetics of Fabry disease. With approximately 270
mutations identified as amenable to this chaperone therapy in
Canada, there are many patients who could potentially benefit from
this new treatment option."
Julia Alton, Executive Director of the Canadian
Fabry Association, stated, “Following Health Canada’s approval of
Galafold, we are delighted that the Fabry community in Canada will
soon have access to the first new Fabry treatment option in nearly
fifteen years. Amicus has partnered with the Fabry community for
more than a decade to incorporate the needs of patients and
physicians into the Galafold development process and we look
forward to the differentiated treatment choice that Galafold will
offer to Fabry patients in Canada who have amenable mutations.”
About Galafold™ and Amenable
MutationsGalafold™ (migalastat) is a first-in-class
chaperone therapy approved in Canada 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 800 known GLA mutations as
“amenable” or “not amenable” to treatment with Galafold. The
Canadian label includes 270 GLA mutations that have been identified
and determined to be amenable in Canada based on the Galafold
Amenability Assay.
Healthcare providers in Canada 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 Canadian label as additional GLA mutations are identified
and tested in the Galafold Amenability Assay.
Important Canadian 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.
- Clinical data supporting the effectiveness of Galafold for the
treatment of Fabry disease patients with amenable mutations are
limited. In clinical trials, individual response to Galafold
treatment varied considerably among patients with amenable
mutations. Patients should be assessed for treatment response or
failure when initiating Galafold, and monitored periodically
thereafter (every 6 months or more frequently) throughout the
treatment
- Galafold is not recommended for use in patients with a
non-amenable mutation. Galafold may result in a net loss of α-Gal A
activity in patients with non-amenable mutations, potentially
worsening the disease condition.
- Galafold is not intended for concomitant use with enzyme
replacement therapy.
- The patient should be advised to carefully adhere to the
recommended dosing regimen of Galafold [one 123 mg migalastat
capsules every other day (QOD)]. A higher dose or shorter dosing
interval may result in a loss of efficacy, potentially worsening
the disease condition
- Galafold should not be used in patients with severe renal
insufficiency, defined as having an estimated glomerular filtration
rate (eGFR) less than 30 mL/min/1.73m2 , due to a significant
increase in the exposure to migalastat and prolonged half-life of
migalastat. This may result in a net loss of α-Gal A activity,
potentially worsening the disease condition.
- The safety and efficacy of GALAFOLDTM in pediatric patients
have not been established
- No dosage adjustments are required in patients with hepatic
impairment or in the elderly population.
- Galafold should not be used by pregnant women and is not
recommended in women of childbearing potential not using
contraception.
- While taking Galafold, effective birth control should be used.
Galafold should not be used in breast-feeding women. It is not
known whether Galafold is excreted in human milk.
- Galafold is contraindicated for use in patients who are
hypersensitive to this drug or to any ingredient in the formulation
or component of the container
- OVERDOSE: General medical care is recommended in the case of
Galafold overdose.
- The most common adverse reaction reported was headache. For a
complete list of adverse reactions, please review the Canadian
Product Monograph.
- Call your doctor for medical advice about side effects.
For further important safety information for Galafold, including
the indications, method of administration, special warnings,
drug interactions and adverse drug reactions, please see the
Canadian Prescribing Information for Galafold available from the
Health Canada website here.
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 clinical development of our product candidates, the
prospects and timing of the potential regulatory and pricing
approval of our product candidates. 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 regulatory and pricing
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: the potential that
results of clinical or preclinical studies indicate that the
product candidates are unsafe or ineffective; the potential that we
may not be successful in commercializing Galafold in Europe and
other geographies, including Canada; and the potential that we may
not be successful in pricing and reimbursement discussions. 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
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