Amicus Therapeutics (Nasdaq: FOLD), a global, patient dedicated
biotechnology company focused on discovering, developing and
delivering novel medicines for rare metabolic diseases, today
announced preliminary 2019 revenue and Galafold (migalastat)
commercial updates.
John F. Crowley, Chairman and Chief Executive Officer of Amicus
Therapeutics stated, “2019 represented another period of
significant growth and adoption for Galafold across all
geographies. We are pleased to announce our commercial performance
surpassed our expectations for the year. This reflects the strong
continued adoption of this novel precision medicine for Fabry
patients with amenable mutations as well as the 90%+ continued
adherence and compliance rates seen with patients. We are also
pleased to have recently received notice of three new marketing
authorizations in key countries that occurred late in the fourth
quarter. These achievements highlight our company’s mission, which
is focused on transforming the lives of people living with rare
diseases, and for Galafold to ensure broad, worldwide access as
soon as possible for all Fabry patients who may benefit.”
Fourth Quarter and Full Year 2019 RevenueGlobal
revenue for Galafold in full-year 2019 was approximately $181
million (preliminary and unaudited) representing a year-over-year
increase of 99% from total revenue of $91 million in 2018 and
exceeded the Company’s updated 2019 guidance of $170 million to
$180 million. Full-year revenue was negatively impacted by
approximately $5 million in foreign currency headwinds. Fourth
quarter Galafold revenue was approximately $54 million (preliminary
and unaudited).
Marketing AuthorizationsIn 2019, Amicus made
significant progress expanding access to Galafold around the globe,
including most recently in Brazil, Colombia and Taiwan. Each of
these countries granted approval for the first-in-class, oral small
molecule precision medicine pharmacological chaperone Galafold
(migalastat), as a monotherapy. Galafold is the first and only oral
precision medicine for Fabry disease approved in these
countries.
In Brazil, Amicus is working to complete the requirements to
launch Galafold in the coming months. As part of the approval
process for rare diseases in the country, patients will initially
be required to go through a court injunction process, which will
take approximately one year before receiving access to medication.
Therefore, the Company expects more meaningful revenue contribution
to begin in 2021 and beyond.
Galafold is approved in over 40 countries around the world,
including the U.S., EU, Japan and others.
Amicus management will present a corporate overview and 2020
guidance at the 38th Annual J.P. Morgan Healthcare Conference in
San Francisco, CA on Tuesday, January 14, 2020 at 8:30 a.m. PT. A
Q&A breakout session will immediately follow the presentation
at 9:00 a.m. PT.
Live audio webcasts of the presentation and breakout session
will be available and can be accessed through the Investors section
of the Amicus Therapeutics corporate website at
http://ir.amicusrx.com/, and will be archived for 90 days.
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, 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 INFORMATION
ADVERSE 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.
EU 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 disorder caused by deficiency of an enzyme
called alpha-galactosidase A (alpha-Gal A), which results from
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 heart, kidneys, and skin. Accumulation
of GL-3 and progressive deterioration of organ function is believed
to lead to the morbidity and mortality of Fabry disease. The
symptoms can be severe, differ from person to person, and begin at
an early age.
About Amicus TherapeuticsAmicus
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 us
on Twitter and LinkedIn.
CONTACTS:
Investors:
Andrew FaughnanDirector, Investor
Relationsafaughnan@amicusrx.com(609) 662-3809
Media:Christopher Byrne Executive Director,
Corporate Communications cbyrne@amicusrx.com (609) 662-2798
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