Amicus Therapeutics Receives the 2024 New Treatment Award for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) at the 20th Annual WORLDSymposium™
February 08 2024 - 7:00AM
Amicus Therapeutics (Nasdaq: FOLD), today announced that
WORLDSymposium™ has awarded Pombiliti™ (cipaglucosidase alfa-atga)
+ Opfolda™ (miglustat) the 2024 New Treatment Award, which
recognizes important achievements in advancing treatments for
lysosomal diseases which have attained regulatory approval.
“We are honored that WORLDSymposium has
recognized Pombiliti and Opfolda with the 2024 New Treatment
Award,” said Bradley Campbell, President and Chief Executive
Officer of Amicus Therapeutics, Inc. “It is a testament to more
than a decade of dedication and commitment from Team Amicus along
with dozens of families, physicians, researchers, and advocates
around the world who worked tirelessly to see this medicine
approved. Their partnership has been pivotal in continuing our
promise to lead the fight in rare diseases, and we share this award
with the entire global Pompe disease community.”
Pombiliti + Opfolda is the first and only
two-component therapy approved by the U.S. Food and Drug
Administration (FDA) for adults with late-onset Pompe disease
weighing ≥40 kg and who are not improving on their current enzyme
replacement therapy (ERT). 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.
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.
This is the second WORLDSymposium™ New Treatment
Award presented to Amicus. Amicus received the award in 2017 for
the first and only oral therapy approved for people living with
Fabry disease.
About Pombiliti +
OpfoldaPombiliti + 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.
U.S. INDICATIONS
AND USAGEPOMBILITI 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).
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. CONTRAINDICATION: 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.
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
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.
PP-AT-ALL-0002-0224
CONTACTS:
Investors: Amicus Therapeutics Andrew
FaughnanVice President, Investor
Relationsafaughnan@amicusrx.com(609) 662-3809
Media: Amicus Therapeutics Diana Moore Head of
Global Corporate Affairs and Communicationsdmoore@amicusrx.com(609)
662-5079
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