Amicus Therapeutics Launches Galafold™ (Migalastat) for Treatment of Fabry Disease in the United Kingdom
February 27 2017 - 7:00AM
National Institute for Health and Care
Excellence (NICE) Publishes Final Guidance Covering Reimbursement
of Galafold for Fabry Disease
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 the United
Kingdom (UK) following the final publication of reimbursement
guidelines by the National Institute for Health and Care Excellence
(NICE) Highly Specialised Technologies Evaluation Committee (EC).
Galafold is reimbursed within the National Health Service (NHS) in
England 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 (alpha-galactosidase A deficiency) and who have an
amenable mutation.
"Galafold is the first oral treatment as well as
the first precision medicine now available in the UK for Fabry
patients 16 years and older who have an amenable mutation,” stated
John F. Crowley, Chairman and Chief Executive Officer of Amicus
Therapeutics, Inc. “Following the final NICE publication, which we
believe reflects the significant value of our oral precision
medicine approach, we are pleased that all appropriate patients
will now have access to Galafold. The finalization of pricing and
reimbursement in the UK is a significant milestone for Amicus as we
navigate the country-by-country processes to launch Galafold
throughout the EU as rapidly as possible.”
Fabry 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. 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).
“We are delighted that Fabry patients with
amenable mutations will now have the option to be prescribed an
oral treatment for the management of their disease,” commented
Christine Lavery, Group Chief Executive of The MPS Society, UK and
President of the Fabry International Network (FIN). “Our mission is
to inform and support patients and families in all aspects related
to their disease, therefore having access to new therapeutic
opportunities is an important consideration and opportunity in a
patient’s journey.”
The European Commission granted full approval
for Galafold on May 30, 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.
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 800 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 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 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 preclinical and clinical development of our product
candidates, the timing and reporting of results from preclinical
studies and clinical trials, the prospects and timing of the
potential regulatory approval of our product candidates,
commercialization plans, financing plans, and the projected cash
position for the Company. 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 authorities, and in
particular the potential goals, progress, timing, and results of
preclinical studies and clinical trials, 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 it may be difficult to enroll
patients in our clinical trials; the potential that regulatory
authorities, including the FDA, EMA, and PMDA, may not grant or may
delay approval for our product candidates; the potential that we
may not be successful in commercializing Galafold in Europe or our
other product candidates if and when approved; the potential that
preclinical and clinical studies could be delayed because we
identify serious side effects or other safety issues; and the
potential that we will need additional funding to complete all of
our studies. Further, the results of earlier preclinical studies
and/or clinical trials may not be predictive of future results.
With respect to statements regarding projections of the Company's
cash position, actual results may differ based on market factors
and the Company's ability to execute its operational and budget
plans. 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, 2015 and Quarterly Report on Form 10-Q for the
quarter ended September 30, 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
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
MWW PR
Sid Dinsay
sdinsay@mww.com
(646) 381-9017
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