Amicus Therapeutics (Nasdaq: FOLD), a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel medicines for rare diseases, today announced that
the Company has proactively taken numerous measures in response to
the novel coronavirus (COVID-19) pandemic to support the rare
disease community globally and to ensure the achievement of its
2020 key strategic priorities. In support of its patients, people,
and programs, a COVID-19 internal Amicus task force chaired by
Chairman & CEO John F. Crowley was established in early March
and meets regularly via video conference to assess COVID-19
developments and their impacts on company plans and strategies.
Our Patients. Amicus is committed to providing
uninterrupted access to medicines for those who are in need of a
treatment.
- Global supply chains for Galafold® are robust and have not been
interrupted. Amicus believes the Company will continue to be able
to supply this oral precision medicine for any and all people
living with Fabry disease who have an amenable variant.
- The Company also has a high degree of confidence that
participants enrolled in all clinical studies for AT-GAA for Pompe
disease, including the Phase 3 PROPEL study, will continue to
receive study drug and that this study will be completed on
schedule.
- The Company has taken steps to reinforce and elevate the
resources provided to patient community partners, which is also
detailed on our corporate website.
Our People. Amicus is aligned with public health
strategies designed to prevent the spread of COVID-19 in order to
protect our global workforce.
- In addition to instituting social distancing efforts, the
Company mandated a work from home policy for all global Amicus
offices as of March 12, 2020.
- Amicus is focused on ensuring the health and wellbeing of its
global workforce and maintaining the safety of all our employees
across the globe. The Amicus Cares internal initiative was launched
last week to respond to the needs of employees during this
unprecedented global health crisis.
- Company executives and medical personnel are also advising and
assisting with local, state and federal authorities in certain
instances.
Our Programs. Amicus remains dedicated to
delivering high-quality medicines for people living with rare
diseases. Toward that end, Amicus has instituted a business
continuity plan that it believes ensures the business will continue
to perform and deliver on its mission for patients and
shareholders. Based on current information, the Company continues
to believe it will achieve its 2020 key strategic priorities,
including:
- Achieve $250 million to $260 million of global product revenue
for Galafold
- Complete Pompe Phase 3 PROPEL study, enroll pediatric studies
and advance manufacturing to support 2021 BLA and MAA of
AT-GAA
- Advance clinical development, manufacturing and regulatory
discussions for CLN6 and CLN3 Batten programs
- Progress Pompe gene therapy towards IND and disclose up to two
additional IND candidates
- Maintain strong financial position
As a leader in the global rare-disease
community, our top priority remains our patients, communities and
employees. We will continue to monitor the overall situation
closely as it applies to all our partners and share updates
appropriately.
About
Galafold Galafold (migalastat)
123 mg capsules is an oral pharmacological chaperone of
alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry
disease in adults who have amenable GLA variants. In
these patients, Galafold works by stabilizing the body’s own
dysfunctional enzyme so that it can clear the accumulation of
disease substrate. Globally, Amicus Therapeutics estimates that
approximately 35 to 50 percent of Fabry patients may have
amenable GLA variants, though amenability rates within
this range vary by geography. Galafold is approved in over 40
countries around the world, including the U.S., EU, U.K, Japan and
others.
U.S. Indications and Usage
Galafold is indicated for the treatment of adults with a confirmed
diagnosis of Fabry disease and an amenable galactosidase alpha gene
(GLA) variant based on in vitro assay data.
This indication is approved under accelerated
approval based on reduction in kidney interstitial capillary cell
globotriaosylceramide (KIC GL-3) substrate. Continued approval for
this indication may be contingent upon verification and description
of clinical benefit in confirmatory trials.
U.S. Important Safety
InformationAdverse Reactions The most
common adverse reactions reported with Galafold (≥10%) were
headache, nasopharyngitis, urinary tract infection, nausea and
pyrexia.
Use in Specific Populations
There is insufficient clinical data on Galafold use in pregnant
women to inform a drug-associated risk for major birth defects and
miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human
milk. Therefore, the developmental and health benefits of
breastfeeding should be considered along with the mother’s clinical
need for Galafold and any potential adverse effects on the
breastfed child from Galafold or from the underlying maternal
condition.
Galafold is not recommended for use in patients
with severe renal impairment or end-stage renal disease requiring
dialysis.
The safety and effectiveness of Galafold have
not been established in pediatric patients.
To report Suspected Adverse Reactions, contact
Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
For additional information about Galafold,
including the full U.S. Prescribing Information, please
visit https://www.amicusrx.com/pi/galafold.pdf.
E.U. and U.K. 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 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.
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, manufacturing and supply plans, financing
plans and the projected revenues and 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, statements regarding corporate goals
and the attainment of such goals, including as they are impacted by
Covid-19 related disruption, are based on current
information. The potential impact on operations from the
Covid-19 outbreak is inherently unknown and cannot be predicted
with confidence and may cause actual results and performance to
differ materially from the statements in this release, including
without limitation, because of the impact on general
political and economic conditions, including as a result of efforts
by governmental authorities to mitigate Covid-19, such as travel
bans, shelter in place orders and third-party business closures and
resource allocations, manufacturing and supply chain disruptions,
limitations on patient access to commercial product or clinical
trial sites and investigational product or other clinical study
disruptions. With respect to statements regarding projections
of the Company's financial outlook, actual results may differ based
on Covid-19 related disruptions and other market factors, including
the factors above, and the Company's ability to execute its
business continuity, 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,
2019. 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
CONTACT:
Andrew FaughnanDirector, Investor
Relationsafaughnan@amicusrx.com (609) 662-3809
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