Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global
biotechnology company focused on developing and commercializing
novel medicines for rare diseases, today announced that the
European Commission (EC) has granted approval for Opfolda®
(miglustat) 65mg capsules, an enzyme stabilizer of cipaglucosidase
alfa, a long-term enzyme replacement therapy for adults with
late-onset Pompe disease. Pombiliti® (cipaglucosidase alfa) was
previously approved by the EC in March 2023. Pompe disease is an
inherited lysosomal disorder caused by deficiency of the enzyme
acid α-glucosidase (GAA). Amicus plans to immediately launch
Pombiliti + Opfolda in Germany and is commencing reimbursement
processes with healthcare authorities in other European countries.
“Late-onset Pompe disease is a rare,
neuromuscular disorder that can have devastating consequences for
patients and their families. The European Commission approval for
Pombiliti and Opfolda is the realization of the work of so many
individuals and teams dedicated to the mission of improving the
lives of people living with Pompe disease. We look forward to
bringing this much needed, new treatment to all adults living in
the EU with late-onset Pompe disease,” said John F. Crowley,
Executive Chairman of Amicus Therapeutics, Inc.
"We are extremely pleased with the EC approval
of Pombiliti and Opfolda, and are grateful to the Pompe community
around the world who have helped advance this therapy. Given the
strength of the label and our launch readiness, we believe
Pombiliti and Opfolda has the potential to become the next standard
of care in this devastating condition by showing that improvement
is possible for people living with late-onset Pompe disease,” said
Bradley Campbell, President and Chief Executive Officer of Amicus
Therapeutics, Inc.
Pombiliti + Opfolda is a unique two-component
therapy. Pombiliti (cipaglucosidase alfa), is a bis-M6P-enriched
rhGAA enzyme, designed for increased uptake into muscle cells. Once
in the cell, Pombiliti can be processed into its most active and
mature form to break down glycogen. Opfolda (miglustat) is an
enzyme stabilizer designed to stabilize the enzyme in the blood.
The EC approval was based on clinical data from the Phase 3 pivotal
study (PROPEL), the only trial in LOPD to study the real-world
population of both ERT-naïve and ERT-experienced participants in a
controlled setting.
“This significant milestone marks the beginning
of broad access to Pombiliti and Opfolda for the LOPD community in
Europe, where there is a high medical need for novel treatment
options,” said Prof. Benedikt Schoser, Professor of Neurology at
Ludwig-Maximilians-University of Munich LMU Department of
Neurology. “In clinical studies, Pombiliti and Opfolda have
exhibited clinically meaningful and positive changes in the key
mobility and respiratory manifestations of this challenging
disease. The EC approval and indication reflect the hope for the
potential of this innovative therapy for people living with Pompe
disease.”
“The Pompe community is greatly appreciative of
Amicus’ long-standing commitment to develop a treatment option to
address the continuing unmet needs of people living with late-onset
disease. Each person deserves alternatives to help them best manage
their condition,” said Tiffany House, President, International
Pompe Association.
In the U.K., the Medicines and Healthcare
products Regulatory Agency (MHRA) regulatory approval is expected
in the third quarter of 2023. The U.S. Food and Drug
Administration’s review is ongoing, and the Company expects
approval in the third quarter of 2023. The FDA previously granted
Breakthrough Therapy designation for Pombiliti + Opfolda.
About
Pombiliti® +
Opfolda®Pombiliti + Opfolda, is a
two-component therapy that consists of cipaglucosidase alfa, 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. In clinical studies, Pombiliti +
Opfolda was associated with demonstrated improvements in both
musculoskeletal and respiratory measures.
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
controlling breathing.
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 diseases. For more
information please visit the company’s website at www.amicusrx.com,
and follow on Twitter and LinkedIn.
Important Safety
InformationPombiliti (cipaglucosidase alfa)
Important Safety Information
Posology and Method of
Administration: Pombiliti must be used in combination with
miglustat 65 mg hard capsules. The recommended dose of Pombiliti is
20 mg/kg of body weight every other week. The Pombiliti infusion
should start 1 hour after taking miglustat capsules.
Paediatric population: The safety and efficacy of
Pombiliti in combination with miglustat therapy in paediatric
patients less than 18 years old have not yet been established. No
data are available.
Contraindications:
Life-threatening hypersensitivity to the active substance, or to
any of the excipients. Contraindication to miglustat.
Anaphylaxis and infusion-associated reactions
(IARs): Serious anaphylaxis and IARs have occurred in some
patients during infusion and following infusion with Pombiliti.
Premedication with oral antihistamine, antipyretics, and/or
corticosteroids may be administered to assist with signs and
symptoms related to IARs experienced with prior enzyme replacement
therapy (ERT) treatment. Reduction of the infusion rate, temporary
interruption of the infusion, symptomatic treatment with oral
antihistamine, or antipyretics, and appropriate resuscitation
measures should be considered to manage serious IARs. If
anaphylaxis or severe allergic reactions occur, infusion should be
immediately paused, and appropriate medical treatment should be
initiated. The current medical standards for emergency treatment of
anaphylactic reactions are to be observed and cardiopulmonary
resuscitation equipment should be readily available. The risks and
benefits of re-administering Pombiliti following anaphylaxis or
severe allergic reaction should be carefully considered, and
appropriate resuscitation measures made available. Risk of
acute cardiorespiratory failure in susceptible patients:
Patients with acute underlying respiratory illness or compromised
cardiac and/or respiratory function may be at risk of serious
exacerbation of their cardiac or respiratory compromise during
infusions. Appropriate medical support and monitoring measures
should be readily available during Pombiliti infusion.
Immune complex-related reactions: Immune
complex-related reactions have been reported with other ERTs in
patients who had high IgG antibody titres, including severe
cutaneous reactions and nephrotic syndrome. If immune
complex-related reactions occur, discontinuation of the
administration of Pombiliti should be considered and appropriate
medical treatment should be initiated. The risks and benefits of
re-administering Pombiliti following an immune complex-related
reaction should be reconsidered for each individual patient.
Contraception in females: Reliable contraceptive
measures must be used by women of childbearing potential during
treatment with Pombiliti in combination with miglustat, and for 4
weeks after discontinuing treatment. Pregnancy:
Pombiliti in combination with miglustat therapy is not recommended
during pregnancy. Breast feeding: It is not known
if Pombiliti and miglustat are secreted in human breast milk. A
decision must be made whether to discontinue breast-feeding or to
discontinue/abstain from Pombiliti in combination with miglustat
therapy taking into account the benefit of breast-feeding for the
child and the benefit of therapy for the woman. Summary of
the safety profile: The most commonly reported adverse
reactions only attributable to Pombiliti were chills (4.0%),
dizziness (2.6%), flushing (2.0%), somnolence (2.0%), chest
discomfort (1.3%), cough, (1.3%), infusion site swelling (1.3%),
and pain (1.3%). Reported serious adverse reactions only
attributable to Pombiliti were urticaria (2.0%), anaphylaxis
(1.3%), pyrexia (0.7%), presyncope (0.7%), dyspnoea (0.7%),
pharyngeal oedema (0.7%), wheezing (0.7%), and hypotension (0.7%).
Refer to SmPC for full list.
Opfolda (miglustat) 65 mg hard capsules
Important Safety Information
Posology and Method of
Administration: Opfolda must be used in combination with
Pombiliti. The recommended dose is to be taken orally every other
week and is based on body weight. Opfolda should be taken
approximately 1 hour but no more than 3 hours before the start of
the Pombiliti infusion. Paediatric population: The
safety and efficacy of Opfolda in combination with Pombiliti
therapy in paediatric patients less than 18 years old have not yet
been established. No data are available.
Contraindications: Hypersensitivity to the active
substance or to any of the excipients. Contraindication to
cipaglucosidase alfa. Food Interaction: Patients
should fast for 2 hours before and 2 hours after taking Opfolda.
Contraception in females: Reliable contraceptive
measures must be used by women of childbearing potential during
treatment with Opfolda in combination with Pombiliti, and for 4
weeks after discontinuing treatment. Pregnancy:
Opfolda crosses the placenta. Opfolda in combination with Pombiliti
therapy is not recommended during pregnancy. Breast
feeding: It is not known if Opfolda and Pombiliti are
secreted in human breast milk. A decision must be made whether to
discontinue breast-feeding or to discontinue/abstain from Opfolda
in combination with Pombiliti therapy taking into account the
benefit of breastfeeding for the child and the benefit of therapy
for the woman. Summary of the safety profile: The
most commonly reported adverse reaction only attributable to
Opfolda 65 mg was constipation (1.3%). Refer to SmPC for full
list.
Forward Looking StatementThis press release
contains "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements relating to data from a global Phase 3 study to
investigate AT-GAA for the treatment of Pompe Disease, the
potential implications on these data for the future advancement and
development of AT-GAA and expectations regarding the regulatory
process in the UK and US. There can be no assurance that the MHRA
or FDA will grant full approval for both components of AT-GAA or
when any such approvals may occur. Words such as, but not limited
to, “look forward to,” “believe,” “expect,” “anticipate,”
“estimate,” “intend,” "confidence," "encouraged," “potential,”
“plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and
“could,” and similar expressions or words identify forward-looking
statements. The forward-looking statements included in this press
release are based on management's current expectations and belief's
which are subject to a number of risks, uncertainties and factors,
including that the Company will not be able to successfully
complete the development of, obtain full global regulatory
approvals for, or successfully manufacture and commercialize AT-GAA
once fully approved in each geography. In addition, all forward
looking statements are subject to the other risks and uncertainties
detailed in our Annual Report on Form 10-K for the year ended
December 31, 2022. As a consequence, actual results may differ
materially from those set forth in this press release. You are
cautioned not to place undue reliance on these forward-looking
statements, which speak only of the date hereof. All
forward-looking statements are qualified in their entirety by this
cautionary statement, and we undertake no obligation to revise this
press release to reflect events or circumstances after the date
hereof.
CONTACT:
Investors: Amicus Therapeutics Andrew
FaughnanVice President, Investor Relationsafaughnan@amicusrx.com+1
(609) 662-3809
Media: Amicus Therapeutics Diana Moore Head of
Global Corporate Communicationsdmoore@amicusrx.com+1 (609)
662-5079
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