Amicus Therapeutics Announces Approval for Galafold™ (Migalastat) for Treatment of Fabry Disease in Australia
August 15 2017 - 4:01PM
First Amicus Medicine and First Oral
Precision Medicine for Fabry Disease in Australia
Amicus Therapeutics (Nasdaq:FOLD) announced that the Australian
Therapeutic Goods Administration (TGA) has approved the oral
precision medicine Galafold 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. Amicus estimates that approximately 35%-50% of
Fabry patients in Australia have an amenable mutation. Following
the TGA approval, Amicus is continuing to work with the Australian
reimbursement authorities to make Galafold available to Australian
patients in a timely manner.
"The approval of Galafold in Australia is a
significant step forward for the Fabry community and reflects our
commitment to providing the first oral precision medicine for Fabry
disease as rapidly as possible to patients throughout the world,”
stated John F. Crowley, Chairman and Chief Executive Officer of
Amicus Therapeutics, Inc. “There has been a tremendous amount of
momentum for the commercial launch and significant progress with
our regulatory submissions and approvals for Galafold. Following
our initial EU approval, this is our second approval through an
independent submission process. We have also secured approvals in
geographies such as Switzerland and Israel that have a regulatory
pathway that accepts the EU approval as the basis for submission
and review. Our next step in Australia is to navigate the pricing
and reimbursement discussions as we continue toward our vision to
deliver Galafold to even more patients in more geographies.”
The Australian TGA approval under the Orphan
Drug program 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 an ongoing long-term extension study. 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
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).
“The Australian approval of Galafold paves the
way for the first new Fabry treatment option in more than a decade,
and provides a clearly differentiated oral precision medicine
option for Fabry patients in Australia who have amenable
mutations,” said Megan Fookes, Managing Director of Fabry
Australia. “We are grateful for Amicus’ commitment to innovation,
patient-focused drug development, and high quality therapies for
the Fabry community.”
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. Marketing applications have also been approved in several
countries outside the EU, including Switzerland, Israel, and now
Australia.
About Galafold™ and Amenable
MutationsGalafold® (migalastat) is a first-in-class
chaperone therapy approved in Australia 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
Australian 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.
Amicus expects to submit additional updates to
the Australian label as additional GLA mutations are identified and
tested in the Galafold Amenability Assay.
Australia 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
the indications, method of administration, special warnings,
drug interactions and adverse drug reactions, please see the
Australian Prescribing Information for Galafold available from the
TGA website at https://www.tga.gov.au/product-information-pi.
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, SD-101 for
Epidermolysis Bullosa (EB), 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 Australia; 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.
FOLD–G
CONTACTS:
Investors/Media:
Amicus Therapeutics
Sara Pellegrino, IRC
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
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