Pompe Disease Treatment Market is expected to reach US$ 4.06 billion by 2033 | Top key players - Sanofi, Amicus Therapeutics, AskBio Inc.

Market Size and Growth:

The Global Pompe Disease Treatment Market Size was valued at approximately US$ 1.28 billion in 2023, with a rise to US$ 1.39 Billion in 2024, forecasting 4.06 Billion in 2033, exhibiting a CAGR of 13.1% during the forecast period (2025-2033). According to DataM Intelligence Report.

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The Pompe Disease Treatment Market encompasses therapies and interventions designed to manage and treat Pompe disease, a rare genetic disorder caused by acid alpha-glucosidase enzyme deficiency. The market includes enzyme replacement therapies, gene therapies, and supportive treatments aimed at improving patient outcomes, reducing disease progression, and enhancing quality of life for affected individuals globally.

United States: Recent Developments of Pompe Disease Treatment Market

✅ September 2025: Aro Biotherapeutics completed enrollment in the Phase 1b trial of ABX1100, a targeted siRNA therapy designed to reduce the treatment burden for late-onset Pompe disease patients by addressing unmet needs beyond current enzyme replacement therapies.​

✅ September 2025: Texas officially added Pompe disease to its newborn screening program, marking a significant advancement in early detection efforts across U.S. states.​

✅ November 2025: Market analyses highlighted ongoing expansion in Pompe disease treatments, driven by increased diagnoses and advancements like the FDA-approved Pombiliti/Opfolda dual therapy for late-onset cases weighing at least 40 kg.​

Europe: Recent Developments of Pompe Disease Treatment Market

✅ September 2025: Reports emerged on gene therapy showing improvements in cardiac and motor function for infantile-onset Pompe disease, based on recent research evaluations.​

✅ October 2025: AskBio presented six updates on gene and cell therapy advancements for Pompe disease at the European Society of Gene and Cell Therapy annual meeting in Seville, Spain.​

✅ October 2025: The European Reference Network hosted a symposium on current Pompe disease treatments and future prospects, featuring discussions by experts like Prof. Benedikt Schoser from Germany.​

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Recent & Latest Regulatory Approvals (2024-2025):

✅ Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) - In June 2025, Japan's Ministry of Health, Labour and Welfare approved this two‐component therapy for adults with late‐onset Pompe disease (LOPD).

✅ In 2025 (September) - data from a four‐year open-label extension (OLE) study (the "PROPEL" study) of Pombiliti + Opfolda showed sustained improvements in muscle function, strength, and biomarkers in treated LOPD patients. This reinforces the long-term efficacy and safety of the combo therapy.

✅ Earlier, in 2023, the U.S. Food and Drug Administration (FDA) had approved Pombiliti + Opfolda for adults with LOPD (≥ 40 kg) who had inadequate response to prior ERT.

✅ The older-generation ERT, Nexviazyme (avalglucosidase alfa), remains approved and used globally (including U.S., Europe, Japan, and other regions) for both infantile-onset and late-onset Pompe disease.

Mergers & Strategic Moves in 2025:

✅ In mid‐2025, BioMarin Pharmaceutical completed acquisition of Inozyme Pharma (for about US$270 million), bringing in their late‐stage enzyme‐replacement therapy asset for a different rare disease. While not a Pompe therapy, this acquisition underscores continued investor and industry interest in rare‐disease enzyme therapies.

✅ In the broader rare-disease/lysosomal‐storage disorder segment, companies including those working on Pompe continue to be identified as key contributors to market growth.

Major Players:

Key companies in the Pompe Disease Treatment market include:

Sanofi
Amicus Therapeutics
AskBio Inc.
Beijing GeneCradle Technology Co., Ltd.
Aro Biotherapeutics Company

Segments Covered in the Pompe Disease Treatment Market:

By Disease Type: Infantile-Onset Pompe Disease, Late-Onset Pompe Disease.

By Treatment Type: Enzyme Replacement Therapy (ERT), Gene Therapy, Substrate Reduction Therapy, Chaperone-Advanced Replacement Therapy (CART), Physical Therapy, Others.

By Route of Administration: Intravenous, Oral, Subcutaneous.

By End-User: Hospitals, Ambulatory Surgical Centers, Homecare Settings, Specialty Clinics, Others.

Regional Analysis for Pompe Disease Treatment Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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Chapter Outline:

⏩ Market Overview: It contains five chapters, as well as information about the research scope, major manufacturers covered, market segments, Pompe Disease Treatment market segments, study objectives, and years considered.

⏩ Market Landscape: The competition in the Global Pompe Disease Treatment Market is evaluated here in terms of value, turnover, revenues, and market share by organization, as well as market rate, competitive landscape, and recent developments, transaction, growth, sale, and market shares of top companies.

⏩ Companies Profiles: The global Pompe Disease Treatment market's leading players are studied based on sales, main products, gross profit margin, revenue, price, and growth production.

⏩ Market Outlook by Region: The report goes through gross margin, sales, income, supply, market share, CAGR, and market size by region in this segment. North America, Europe, Asia Pacific, Middle East & Africa, and South America are among the regions and countries studied in depth in this study.

⏩ Market Segments: It contains the deep research study which interprets how different end-user/application/type segments contribute to the Pompe Disease Treatment Market.

⏩ Market Forecast: Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

⏩ Research Findings: This section of the report showcases the findings and analysis of the report.

⏩ Conclusion: This portion of the report is the last section of the report where the conclusion of the research study is provided.

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Frequently asked questions:

➠ What is the global sales value, production value, consumption value, import and export of Pompe Disease Treatment market?

➠ Who are the global key manufacturers of the Pompe Disease Treatment Industry? How is their operating situation (capacity, production, sales, price, cost, gross, and revenue)?

➠ What are the Pompe Disease Treatment market opportunities and threats faced by the vendors in the global Pompe Disease Treatment Industry?

➠ Which application/end-user or product type may seek incremental growth prospects? What is the market share of each type and application?

➠ What focused approach and constraints are holding the Pompe Disease Treatment market?

➠ What are the different sales, marketing, and distribution channels in the global industry?

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