Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global
biotechnology company focused on developing and commercializing
novel medicines for rare diseases, today announced that the
Medicines and Healthcare products Regulatory Agency (MHRA) of the
United Kingdom (U.K.) has granted marketing authorizations for
Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults
living with late-onset Pompe disease (acid α-glucosidase [GAA]
deficiency). The indication for Pombiliti is a long-term enzyme
replacement therapy (ERT) used in combination with miglustat for
adults with late-onset Pompe disease (LOPD). The indication for
Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term
enzyme replacement therapy for adults with LOPD.
In addition, the National Institute for Health
and Care Excellence (NICE) issued final guidance recommending
reimbursement of Pombiliti + Opfolda for use within the National
Health Service (NHS) in England and Wales. As stated in the
guidance, NICE concluded that the cost-effectiveness estimates for
Pombiliti + Opfolda showed a positive net health benefit and
recommended Pombiliti + Opfolda for adults with LOPD as first line
and later lines of therapy.
Prior to Great Britain (GB) approval, Pombiliti
+ Opfolda was granted an Innovation Passport under
the Innovative Licensing and Access Pathway (ILAP), a
Priority Innovative Medicines designation, as well as a positive
scientific opinion under the Early Access to Medicines Scheme
(EAMS). This accelerated the time to market and enabled healthcare
professionals to prescribe the treatment prior to marketing
authorization based on clinical factors for patients with a clear
unmet need.
“The MHRA approvals for Pombiliti and Opfolda
are a major step forward for adults in the U.K. living with
late-onset Pompe who are seeking new treatments. We are grateful to
the global Pompe community who have helped advance this therapy,
especially the patients, families, and physicians who participated
in our clinical studies,” stated Bradley Campbell, President and
Chief Executive Officer of Amicus Therapeutics, Inc. “The speed in
which NICE recommended reimbursement of Pombiliti and Opfolda is
reflective of the U.K.’s Innovative Licensing and Access Pathway,
the data behind Pombiliti and Opfolda, the strong collaboration
with the reimbursement authorities, and the Amicus commitment to
bring this therapy to those living with Pompe disease as quickly as
possible. I am proud of Amicus’ relentless commitment toward
ensuring patient access to our innovative therapies, and we are
working as quickly as possible to make Pombiliti and Opfolda
commercially available.”
Late-onset Pompe disease (LOPD) 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 muscles and other tissues. Disease severity ranges
on a spectrum, but predominant manifestations are skeletal muscle
weakness and progressive respiratory involvement.
Prof. Mark Roberts, Consultant Neurologist at
the Greater Manchester Neurosciences Unit at Salford Royal NHS
Foundation Trust stated, “From the positive uptake of the Early
Access to Medicines Scheme, we have already seen the impact that
this treatment is having for patients. Having widespread access to
this treatment is an exciting development for the Pompe community,
giving HCPs and patients a new option that exhibits a novel mode of
action.”
The MHRA and NICE decisions were based on
clinical data from the Phase 3 pivotal study (PROPEL), the only
trial in LOPD to study both ERT-naïve and ERT-experienced
participants in a controlled setting.
Allan Muir, Chair of the Board of Trustees of
Pompe Support Network, stated, “Pompe disease is a rare disease,
one that extensively affects all aspects of life for an individual
and their family, friends, and colleagues. We are very grateful to
companies such as Amicus Therapeutics that seek to make meaningful
differences for our small community and we welcome new treatments
that bring further choice to patients.”
Val Buxton, Chief Executive, Association for
Glycogen Storage Diseases, stated, “Being able to gain rapid access
to effective new therapies via the Early Access to Medicines Scheme
has had a meaningful impact on many people affected by Pompe. This
approval has provided a much-needed new therapy option for the
Pompe community.”
Pombiliti + Opfolda has demonstrated fulfilment
of the Great Britain orphan designation criteria and will be added
to the Orphan Register held by the MHRA. Both will benefit from 10
years of market exclusivity in respect of similar medicinal
products in the approved orphan indication.
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.
Important Safety
InformationPombiliti (cipaglucosidase alfa)
Important Safety Information
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.Contraindications:
Life-threatening hypersensitivity to the active substance, or to
any of the excipients. Contraindication to miglustat.
Special warnings and precautions for use:
Pombiliti must be used in combination with Opfolda 65 mg hard
capsules. Anaphylaxis and infusion-associated reactions
(IARs): Serious anaphylaxis and IARs have occurred in some
patients during infusion and following infusion with Pombiliti. 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.
Opfolda (miglustat) 65 mg hard capsules
Important Safety Information
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. Contraindications: Hypersensitivity to the
active substance or to any of the excipients. Contraindication to
cipaglucosidase alfa. Special warnings and precautions for
use: Opfolda 65 mg hard capsules must be used in
combination with Pombiliti. Food Interaction:
Patients should fast for 2 hours before and 2 hours after taking
Opfolda.
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 us on Twitter and
LinkedIn.
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 the anticipated commercial launch of
Pombiliti and Opfolda in the UK. There can be no assurance
the launch will be commercially successful and that pricing and
reimbursement negotiations will be successful outside of England
and Wales. 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 Pombiliti and Opfolda. 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.
CONTACTS:
Investors: Andrew Faughnan Vice President,
Investor Relations afaughnan@amicusrx.com+1 (609) 662-3809
Media: Diana Moore Head of Global Corporate
Communications dmoore@amicusrx.com+1 (609) 662-5079
FOLD–G
NP-AT-ALL-00020823 August 2023
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