Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company
focused on discovering, developing and delivering novel medicines
for rare metabolic diseases, today announced that Galafold®
(migalastat) was awarded the 2018 UK Prix Galien Medal for
Innovative Product. The Prix Galien is awarded to companies who
have made significant advances in pharmaceutical research, and is
regarded as the highest accolade for biomedical research and
development.
John F. Crowley, Chairman and Chief Exeutive
Officer of Amicus Therapeutics, Inc., stated, “We are honored to
receive the prestigious UK Prix Galien medal for Galafold, the
first ever orphan drug to win in the Innovative Product category.
We believe that this award embodies the efforts among our team at
Amicus, together with patients, physicians and the broader Fabry
community, to globally develop and deliver Galafold as a medicine
for certain individuals who are diagnosed with Fabry disease and
who have an amenable GLA mutation, or variant. The Prix Galien
awards are considered by many to be the industry’s equivalent of a
Nobel Prize, and we are grateful to the Galien Foundation for this
very important recognition.”
“The 2018 Awards yet again confirmed the depth and diversity of
innovation in UK life sciences. The success of products for rare
disease – which, for the first time, dominated the shortlist of
finalists – shows that innovation is not confined to Big Pharma or
treatments for major headline conditions," said Karen Westaway,
Chief Executive of ValueBase, owners of Prix Galien’s UK
franchise.
Galafold is approved in the European Union (EU),
U.S., Australia, Canada, Israel, Japan, South Korea and
Switzerland.
About Prix GalienThe Prix Galien Awards were
founded in France in 1970 to celebrate the endeavours of
scientists, researchers and industry to develop innovations that
advance the treatment of human disease. The Prix Galien Awards are
held in 17 countries. The UK Prix Galien Awards are held every two
years at the Houses of Parliament, London. Since its inception in
1990, just 36 products have won a coveted UK Prix Galien medal,
including some of the industry’s most iconic brands. The 2018 UK
Prix Galien awards recognized innovation across four categories:
Innovative Product, Orphan Product, Real World Evidence and Medical
Device.
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 Australia, Canada, European
Union, Israel, Japan, South Korea, Switzerland and the U.S.
U.S. INDICATIONS AND USAGEGalafold 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 REACTIONSThe most common adverse
reactions reported with Galafold (≥10%) were headache,
nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONSThere 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.
About Fabry DiseaseFabry disease is a rare,
progressive genetic disorder characterized by a defective gene
(GLA) that causes an enzyme deficiency. This enzyme is responsible
for breaking down disease substrate that, when deficient in
patients with Fabry disease, builds up in the kidneys, which is one
of the organ systems impacted by Fabry disease.
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 us 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.
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. 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, 2017 as well as our Quarterly
Report on Form 10-Q for the quarter September 30, 2018 filed
November 6, 2018 with the Securities and Exchange Commission. 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.
NP-GA-ALL-00021218
CONTACTS:
Investors/Media:Amicus
TherapeuticsSara Pellegrino, IRCVice President, Investor Relations
& Corporate Communicationsspellegrino@amicusrx.com(609)
662-5044
Media:Pure CommunicationsJennifer Paganelli
jpaganelli@purecommunications.com (347) 658-8290
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