Amicus Therapeutics Announces Approval of Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) in Japan

By: Amicus Therapeutics, Inc. via GlobeNewswire

June 25, 2025 at 07:00 AM EDT

PRINCETON, N.J., and TOKYO, June 25, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for the treatment of adult patients with late-onset Pompe disease (LOPD).

"We are delighted that we will now be able to offer a compelling new treatment option to patients living with late-onset Pompe disease in Japan. We are grateful to the MHLW and to Japan’s Pompe community, including the patients, families, and physicians who participated in our clinical studies, for their collaboration,” said Bradley Campbell, President and Chief Executive Officer, Amicus Therapeutics, Inc.

Pombiliti + Opfolda is a 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. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.

The MHLW approval for Pombiliti + Opfolda was based on clinical data from the Phase 3 pivotal study (PROPEL). PROPEL is the only trial in LOPD to study the real-world population of both ERT-naïve and ERT-experienced participants in a controlled setting.

With this approval, Pombiliti + Opfolda is now approved in the U.S., E.U., U.K., Canada, Australia, Switzerland and Japan.

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.

U.S. 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).

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 Late-Onset Pompe Disease
Late-onset Pompe 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. 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.

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 X and LinkedIn.

CONTACT:

Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809

Media:
Amicus Therapeutics
Diana Moore
Vice President, Corporate Communications
dmoore@amicusrx.com
(609) 662-5079

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