Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S.
Food and Drug Administration (FDA) has approved Pombiliti™
(cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules.
This two-component therapy is indicated for adults living with
late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not
improving on their current enzyme replacement therapy (ERT).
Late-onset Pompe disease is a rare,
debilitating, and life-threatening lysosomal disorder caused by a
deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced
levels of GAA lead to the accumulation of the substrate glycogen in
the lysosomes of muscle cells and glycogen buildup causes muscle
damage. Disease severity ranges across a spectrum, with predominant
manifestations such as skeletal muscle weakness and progressive
respiratory involvement.1
Pombiliti + Opfolda is a unique two-component
therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA)
naturally expressed with high levels of bis-M6P (Mannose
6-Phosphate), designed for increased uptake into muscle cells. Once
in the cell, Pombiliti can be properly processed into its most
active and mature form to break down glycogen. Opfolda is an enzyme
stabilizer designed to stabilize the enzyme in the blood.
“Today’s FDA approval of Pombiliti and Opfolda
is a testament to the power of science, medicine, and our
passionate determination to improve the lives of people living with
Pompe disease. This approval embodies our Amicus spirit, passion,
and resilience and is a very meaningful step for the Pompe
community. I am just so immensely proud of our team, and so very
grateful to everyone who has worked to bring this medicine to this
approval. Most especially to all of the people living with Pompe
around the world,” said John F. Crowley, Executive Chairman of
Amicus Therapeutics, Inc.
Bradley Campbell, President and Chief Executive
Officer of Amicus Therapeutics, Inc., stated, “The FDA approval of
Pombiliti and Opfolda is a major milestone for Amicus. We are
grateful to the Pompe community, particularly the patients,
caregivers, families, researchers, and physicians who have
contributed to the development process through their commitment to
our clinical studies. Today’s approval is also a testament to Team
Amicus’ extraordinary dedication to patients and our ability to
execute on our vision to bring new therapies to the rare disease
community. Our highly experienced team is ready to launch this
medicine in the U.S., and we look forward to rapidly bringing this
new treatment regimen to all eligible adults living with late-onset
Pompe disease who are not improving on their current ERT.”
The FDA approval was based on clinical data
observed from the Phase 3 pivotal study (PROPEL), the only trial in
LOPD to study ERT-experienced participants in a controlled
setting.
“The Pompe community continues to face unmet
need and limited treatment options. This two-component therapy is
an important new treatment for those adults living with late-onset
Pompe disease and not improving on current therapies. I am
encouraged by the evidence generated over many years of clinical
research studying this therapy for ERT-experienced patients living
with late-onset Pompe disease,” said Tahseen Mozaffar, MD, Director
of the Division of Neuromuscular Diseases in the Department of
Neurology at the School of Medicine at UC Irvine and Director of
the UC Irvine ALS and Neuromuscular Center, as well as an
investigator for the PROPEL study.
“Today’s FDA approval is an extremely important
step and acknowledges the potential of Pombiliti and Opfolda,” said
Priya Kishnani, MD, Professor of Pediatrics and Chief of Medical
Genetics at Duke University School of Medicine and an investigator
for the PROPEL study. “I am grateful that eligible patients with
late-onset Pompe disease in the U.S. will now have access to
additional treatment options.”
“The FDA approval of Pombiliti and Opfolda
represents a long-awaited day for people living with late-onset
Pompe disease and advocating for additional therapeutic options,”
said Tiffany House, President, Acid Maltase Deficiency Association.
“Amicus’ long-standing commitment to the Pompe community and rare
disease research has led to the development of an important therapy
for the Pompe community because patients will now have
options.”
Amicus Therapeutics will launch Pombiliti +
Opfolda immediately in the U.S. The FDA previously granted
Breakthrough Therapy designation for Pombiliti + Opfolda. Pombiliti
+ Opfolda has also been approved for the treatment of adults with
LOPD in the European Union and the United Kingdom.
Amicus Assist® provides support to patients and
caregivers in the U.S. and can help patients access their
medication and identify possible sources of financial assistance.
For more information on Amicus Assist, visit the Amicus Assist
website at amicusassist.com, or please call +1-833-AMICUS-A
(+1-833-264-2872).
SAFETY INFORMATION
HYPERSENSITIVITY REACTIONS INCLUDING
ANAPHYLAXIS: Appropriate medical support measures, including
cardiopulmonary resuscitation equipment, should be readily
available. If a severe hypersensitivity reaction occurs, POMBILITI
should be discontinued immediately and appropriate medical
treatment should be initiated. INFUSION-ASSOCIATED REACTIONS
(IARs): If severe IARs occur, immediately discontinue POMBILITI and
initiate appropriate medical treatment. RISK OF ACUTE
CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients
susceptible to fluid volume overload, or those with acute
underlying respiratory illness or compromised cardiac or
respiratory function, may be at risk of serious exacerbation of
their cardiac or respiratory status during POMBILITI infusion. See
PI for complete Boxed Warning. CONTRADICATION: POMBILITI
in combination with Opfolda is contraindicated in
pregnancy. EMBRYO-FETAL TOXICITY: May cause
embryo-fetal harm. Advise females of reproductive potential of the
potential risk to a fetus and to use effective contraception during
treatment and for at least 60 days after the last dose.
Adverse Reactions: Most common adverse reactions ≥ 5% are
headache, diarrhea, fatigue, nausea, abdominal pain, and
pyrexia. Please see full PRESCRIBING INFORMATION, including
BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga)
LINK and full Prescribing Information for
OPFOLDA (miglustat)
LINK.
Conference
Call and
WebcastAmicus Therapeutics will host a conference
call and audio webcast today, September 28, 2023, at 12:00 p.m. ET
to discuss the FDA approval. Participants and investors interested
in accessing the call by phone will need to register using the
online registration form. After registering, all phone participants
will receive a dial-in number along with a personal PIN to access
the event.
A live audio webcast and related presentation
materials can also be accessed via the Investors section of the
Amicus Therapeutics corporate website at ir.amicusrx.com. Web
participants are encouraged to register on the website 15 minutes
prior to the start of the call. An archived webcast and
accompanying slides will be available on the Company's website
shortly after the conclusion of the live event.
About Pompe DiseasePompe
disease is an inherited lysosomal disorder caused by
deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or
absent levels of GAA lead to accumulation of glycogen in cells,
which is believed to result in the clinical manifestations of Pompe
disease. Pompe disease ranges from a rapidly deteriorating
infantile form with significant impact to heart function, to a more
slowly progressive, late-onset form primarily affecting skeletal
muscle and progressive respiratory involvement. Late-onset Pompe
disease can be severe and debilitating with progressive muscle
weakness throughout the body that worsens over time, particularly
skeletal muscles and muscles that control
breathing.1
About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy
that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched
rhGAA that facilitates high-affinity uptake through the M6P
receptor while retaining its capacity for processing into the most
active form of the enzyme, and the oral enzyme stabilizer,
miglustat, that’s designed to reduce loss of enzyme activity in the
blood.
INDICATIONS AND
USAGE
POMBILITI in combination with OPFOLDA is
indicated for the treatment of adult patients with late-onset Pompe
disease (lysosomal acid alpha-glucosidase [GAA] deficiency)
weighing ≥40 kg and who are not improving on their current enzyme
replacement therapy (ERT).
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
diseases. With extraordinary patient focus, Amicus Therapeutics is
committed to advancing and expanding a pipeline of cutting-edge,
first- or best-in-class medicines for rare metabolic diseases.
Further information about the Company can be found at:
www.amicusrx.com, and can be followed 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 approval and
commercialization plans for Pombiliti + Opfolda 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 Pombiliti + Opfolda in the United States, the
potential that public and commercial payors will not reimburse
Pombiliti + Opfolda, 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,
2022, as well as our Quarterly Report on Form 10-Q for the quarter
ended June 30, 2023, filed 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.
CONTACTS:
Investors: Amicus Therapeutics Andrew
FaughnanVice President, Investor
Relationsafaughnan@amicusrx.com(609) 662-3809
Media: Amicus Therapeutics Diana Moore Head of
Global Corporate Communicationsdmoore@amicusrx.com(609)
662-5079
- Kishnani PS, Steiner RD, Bali D, et
al. Pompe disease diagnosis and management guideline. Genet Med
2006; 8: 267–88.
FOLD-G
PP-AT-US-0008-0623
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/a70f1858-5aa5-45ec-8e4b-f7092cff817a
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