Amicus Therapeutics Announces Approval of Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) in Japan
Amicus Therapeutics (Nasdaq: FOLD) has received approval from Japan's Ministry of Health, Labour and Welfare (MHLW) for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) to treat adult patients with late-onset Pompe disease (LOPD). The approval was based on the Phase 3 PROPEL study, which uniquely evaluated both ERT-naïve and ERT-experienced participants.
This two-component therapy consists of Pombiliti, a recombinant human GAA enzyme designed for increased muscle cell uptake, and Opfolda, an enzyme stabilizer. The treatment is now approved across major markets including the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan.
Amicus Therapeutics (Nasdaq: FOLD) ha ottenuto l'approvazione dal Ministero della Salute, del Lavoro e del Welfare giapponese (MHLW) per Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) nel trattamento di pazienti adulti con malattia di Pompe a esordio tardivo (LOPD). L'approvazione si basa sullo studio di Fase 3 PROPEL, che ha valutato in modo unico sia partecipanti naïve alla terapia enzimatica sostitutiva (ERT) sia quelli già trattati.
Questa terapia a due componenti comprende Pombiliti, un enzima GAA umano ricombinante progettato per un maggiore assorbimento nelle cellule muscolari, e Opfolda, uno stabilizzatore dell'enzima. Il trattamento è ora approvato nei principali mercati, tra cui Stati Uniti, Unione Europea, Regno Unito, Canada, Australia, Svizzera e Giappone.
Amicus Therapeutics (Nasdaq: FOLD) ha recibido la aprobación del Ministerio de Salud, Trabajo y Bienestar de Japón (MHLW) para Pombiliti® (cipaglucosidasa alfa) + Opfolda® (miglustat) para tratar a pacientes adultos con enfermedad de Pompe de inicio tardío (LOPD). La aprobación se basó en el estudio de Fase 3 PROPEL, que evaluó de manera única a participantes tanto sin tratamiento previo con terapia de reemplazo enzimático (ERT) como con experiencia en ERT.
Esta terapia de dos componentes consiste en Pombiliti, una enzima humana GAA recombinante diseñada para una mayor absorción en las células musculares, y Opfolda, un estabilizador enzimático. El tratamiento ahora está aprobado en los principales mercados, incluyendo EE.UU., UE, Reino Unido, Canadá, Australia, Suiza y Japón.
Amicus Therapeutics (나스닥: FOLD)가 일본 후생노동성(MHLW)으로부터 Pombiliti® (시파글루코시다제 알파) + Opfolda® (미글루스타트)를 성인 후기 발병 폼페병(LOPD) 환자 치료에 승인받았습니다. 이번 승인은 ERT 치료 경험이 없는 환자와 경험이 있는 환자를 모두 독특하게 평가한 3상 PROPEL 연구를 기반으로 합니다.
이 2성분 치료법은 근육 세포 흡수를 높이도록 설계된 재조합 인간 GAA 효소인 Pombiliti와 효소 안정제인 Opfolda로 구성되어 있습니다. 이 치료법은 현재 미국, 유럽연합, 영국, 캐나다, 호주, 스위스, 일본 등 주요 시장에서 승인되었습니다.
Amicus Therapeutics (Nasdaq : FOLD) a obtenu l'approbation du Ministère japonais de la Santé, du Travail et du Bien-être (MHLW) pour Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) afin de traiter les patients adultes atteints de la maladie de Pompe à début tardif (LOPD). Cette approbation repose sur l'étude de phase 3 PROPEL, qui a évalué de manière unique des participants naïfs et expérimentés en thérapie enzymatique substitutive (ERT).
Cette thérapie à deux composants comprend Pombiliti, une enzyme GAA humaine recombinante conçue pour une meilleure absorption par les cellules musculaires, et Opfolda, un stabilisateur enzymatique. Le traitement est désormais approuvé sur les principaux marchés, notamment les États-Unis, l'Union européenne, le Royaume-Uni, le Canada, l'Australie, la Suisse et le Japon.
Amicus Therapeutics (Nasdaq: FOLD) hat die Zulassung vom japanischen Ministerium für Gesundheit, Arbeit und Wohlfahrt (MHLW) für Pombiliti® (Cipaglucosidase alfa) + Opfolda® (Miglustat) zur Behandlung erwachsener Patienten mit spät einsetzender Pompe-Krankheit (LOPD) erhalten. Die Zulassung basiert auf der Phase-3-Studie PROPEL, die sowohl ERT-naive als auch ERT-erfahrene Teilnehmer einzigartig evaluiert hat.
Diese Zweikomponenten-Therapie besteht aus Pombiliti, einem rekombinanten humanen GAA-Enzym, das für eine verbesserte Aufnahme in Muskelzellen entwickelt wurde, und Opfolda, einem Enzymstabilisator. Die Behandlung ist nun in wichtigen Märkten wie den USA, der EU, Großbritannien, Kanada, Australien, der Schweiz und Japan zugelassen.
- Expansion into Japanese market with MHLW approval for Pombiliti + Opfolda
- Treatment now approved in all major global markets
- Only LOPD trial (PROPEL) to study both ERT-naïve and ERT-experienced patients in controlled setting
- Treatment carries risks of hypersensitivity reactions including anaphylaxis
- Potential for serious infusion-associated reactions requiring discontinuation
- Contraindicated in pregnancy with risk of embryo-fetal toxicity
Insights
Japan's approval of Pombiliti + Opfolda expands Amicus's global reach for LOPD treatment, strengthening revenue potential and market position.
The approval of Pombiliti + Opfolda by Japan's Ministry of Health represents a significant market expansion for Amicus Therapeutics in their rare disease portfolio. This two-component therapy for late-onset Pompe disease (LOPD) has now secured approval in seven major markets including the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan.
What makes this approval particularly valuable is Japan's standing as the world's third-largest pharmaceutical market. The Japanese regulatory approval indicates the treatment met the country's stringent efficacy and safety standards, which builds credibility for the therapy globally.
This approval leverages data from the Phase 3 PROPEL study, which was distinctive in its inclusion of both enzyme replacement therapy (ERT)-naïve and ERT-experienced patients. This real-world population approach in the clinical trial design enhances the therapy's marketability, as it demonstrates effectiveness across a broader patient spectrum.
For Amicus's business model, each geographic expansion is crucial because rare disease treatments like this depend on maximizing global reach to achieve commercial viability. With LOPD affecting approximately 1 in 40,000 people, the addressable patient population in any single country is limited, making multi-market presence essential for revenue growth.
The treatment's mechanism of action offers a differentiated approach compared to existing therapies. The high bis-M6P design for improved muscle cell uptake, combined with an enzyme stabilizer, addresses a fundamental challenge in Pompe disease treatment - getting sufficient active enzyme into affected muscle cells.
Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Now Approved in Japan for Adults with Late-onset Pompe Disease (LOPD)
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 ≥
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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