First Precision and First Oral Medicine
for Fabry Disease Approved for 348 Amenable GLA
Variants
Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S.
Food and Drug Administration (FDA) has granted accelerated approval
of Galafold™ (migalastat) 123 mg capsules. Galafold is an
oral, precision medicine 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.
Galafold was approved under the Subpart H Accelerated Approval
pathway based on reduction in kidney interstitial capillary cell
globotriaosylceramide (KIC GL-3) substrate. As a condition of
accelerated approval, Amicus Therapeutics will continue to study
Galafold in a confirmatory Phase 4 program. Galafold is a capsule
taken once every other day, at the same time of day. The FDA has
approved Galafold for 348 amenable GLA variants. Galafold is the
first oral medicine for Fabry disease, and the first new therapy
approved to treat Fabry disease in the United States in more than
15 years.
John F. Crowley, Chairman and Chief Executive Officer of Amicus
Therapeutics, Inc., stated, “This FDA approval of Galafold is a
transformative moment for people in the U.S. living with Fabry
disease, as it gives adult patients with amenable GLA variants a
new treatment option for the first time in more than 15 years. The
Fabry disease community has had an active voice in every stage of
development of this medicine. We are grateful to this wonderful and
passionate community, particularly the patients and physicians who
have made this research possible through their participation in the
clinical trials, as well as to the U.S. regulators and our
ever-persistent and dedicated Amicus team. This achievement
highlights our company’s mission, which is focused on transforming
the lives of people living with rare diseases. With our new and
highly motivated U.S. leadership team, we are poised to make
Galafold available to as many appropriate patients as
possible.”
Fabry 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. In the U.S., it is estimated that more
than 3,000 people are living with Fabry disease, and an estimated
more than 50 percent of these diagnosed patients are currently
untreated. Globally, it is estimated that 35 to 50 percent of Fabry
disease patients may have an amenable GLA variant.
“People with rare diseases often have limited treatment options,
so research leading to new therapies is critical. As a physician
and researcher, I treated the first patient ever to receive
Galafold in a clinical trial. Today, many years later, I am pleased
that more patients will have access to this precision medicine,”
said Raphael Schiffmann, M.D., M.H.Sc., Director, Baylor Scott
& White Research Institute, Kimberly H. Courtwright and Joseph
W. Summers Institute of Metabolic Disease – Dallas.
The FDA approval was based on data from a Phase 3 pivotal Fabry
disease study in treatment-naïve patients (Study 011, or FACETS),
which demonstrated the efficacy of Galafold, including a reduction
in the amount of damaging disease substrate accumulated in the
kidney capillaries. The most common side effects related to
Galafold were headache, nasopharyngitis, urinary tract infection,
nausea and pyrexia.
“Today is a long-awaited day of celebration for all of us living
with and advocating for people with Fabry disease, especially those
who have participated in the development of Galafold in the United
States,” said Jack Johnson, Founder and Executive Director, Fabry
Support & Information Group. “With the FDA approval of
Galafold, certain members of the U.S. Fabry disease patient
community finally have a second treatment option. Through their
unwavering commitment and scientific innovation, Amicus has
provided a much-needed new treatment option for many Fabry
patients.”
Amicus Therapeutics will launch Galafold immediately, and will
begin shipping to a limited distribution network in the coming
week. Amicus Assist, a service that will provide product
assistance and support to patients to help gain access to Amicus
Therapeutics’ medications, will also be initiated immediately; for
more information please call +1-833-AMICUS-A (+1-833-264-2872).
With today’s approval, patients in 20 countries worldwide have
reimbursable access to Galafold. Galafold is approved in Australia,
Canada, European Union, Israel, Japan, South Korea, Switzerland and
the U.S. Galafold is pending approval in Taiwan.
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast Monday, August 13, 2018, at 8:00 a.m. ET to discuss
the FDA approval. Interested participants and investors may access
the conference call by dialing +1-877-303-5859 (U.S./Canada) or
+1-678-224-7784 (international), conference ID: 9782489.
An audio webcast can also be accessed via the Investors section
of the Amicus Therapeutics corporate website at
http://ir.amicusrx.com/, and will be archived for 30 days. Web
participants are encouraged to go to the website 15 minutes prior
to the start of the call to register, download and install any
necessary software. A telephonic replay of the call will be
available for seven days beginning at 12:00 p.m. ET on August 13,
2018. Access numbers for this replay are +1-855-859-2056
(U.S./Canada) and +1-404-537-3406 (international); conference ID:
9782489.
About Galafold GalafoldTM (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.
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 TherapeuticsAmicus Therapeutics
(Nasdaq: FOLD) is a global, patient-centric biotechnology company
focused on discovering, developing and delivering novel
high-quality medicines for people living with rare metabolic
diseases. The cornerstone of the Amicus portfolio is Galafold, an
oral precision medicine for people living with Fabry disease who
have amenable GLA variants. The lead biologics program in the
Amicus Therapeutics pipeline is AT-GAA, an investigational therapy
for Pompe disease. 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.
Forward-Looking Statements This
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to approval and commercialization plans for Galafold in
the United States. 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, 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 we may not be successful in
commercializing Galafold in the United States, the potential that
public and commercial payors will not reimburse Galafold, the
potential that we may not be able to manufacture or supply
sufficient commercial products; and the potential that we will need
additional funding to complete all of our commercialization and
manufacturing activities. 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 ended June 30, 2018
filed August 7, 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.
A video accompanying this announcement is available
at http://www.globenewswire.com/NewsRoom/AttachmentNg/673926ca-4565-41cf-9f11-f1171f59fd90
Logos accompanying this announcement are available at
http://www.globenewswire.com/NewsRoom/AttachmentNg/0e1eab28-2505-45f8-9660-b1e7d3d9aaba and http://www.globenewswire.com/NewsRoom/AttachmentNg/e5aa5f58-56a0-428c-8b7f-c214e103a3f5
A PDF accompanying this announcement is available
at http://resource.globenewswire.com/Resource/Download/167e6127-f1ea-45ba-ac0a-a28bf35b6fdc
CONTACTS:
Investors:Amicus Therapeutics Andrew Faughnan
Associate Director, Investor Relations afaughnan@amicusrx.com
(609) 662-3809
Media / Multimedia Assets:Pure
CommunicationsJennifer
Paganellijpaganelli@purecommunications.com(347) 658-8290
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