Press release
Pompe Disease Market Expansion Continues, with Forecast Valuation of $2.31 Billion by 2029
Use code ONLINE20 to get 20% off on global market reports and stay ahead of tariff changes, macro trends, and global economic shifts.Pompe Disease Market Size Valuation Forecast: What Will the Market Be Worth by 2025?
The valuation of the Pompe disease market has observed consistent upward movement across recent years, projected to increase from a figure of $1.74 billion in 2024 to $1.8 billion the following year, reflecting a compound annual growth rate of 3.3%. This expansion observed during the preceding timeframe is primarily attributable to factors such as heightened recognition and improved diagnostic procedures, the benefits conferred by orphan drug status and associated incentives, worldwide collaborative efforts and ongoing research endeavors, alongside the dedicated work of patient advocacy organizations and support networks.
Pompe Disease Market Size Forecast: What's the Projected Valuation by 2029?
Forecasting robust expansion, the market pertaining to Pompe disease is projected to achieve a valuation of $2.31 billion by the year 2029, exhibiting a compound annual growth rate (CAGR) of 6.5% throughout the subsequent period. This predicted uptrend is underpinned by several factors, including ongoing efforts in research and development, the proliferation of screening initiatives for newborns, progress being made in gene therapy modalities, augmented worldwide funding allocated to rare ailments, a focus on creating medications tailored to individual patients, the broadening scope of clinical investigations and associated research endeavors, and a market pivot towards individualized treatment methodologies. Key developments anticipated to shape this timeframe involve novel product introductions, improvements in underlying technology, the rollout of dedicated patient assistance schemes, increased utilization of remote healthcare for managing patient care, and partnerships aimed at broadening program accessibility.
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What Are the Drivers Transforming the Pompe Disease Market?
The mounting incidence of Pompe disease is anticipated to fuel the expansion within the Pompe disease market. This condition, alternatively termed glycogen storage disease type II or acid maltase deficiency, represents an infrequent, inherited ailment where glycogen, a complex carbohydrate, accumulates within numerous bodily tissues, principally impacting muscles and various organs. Current therapeutic interventions for Pompe disease encompass protein replacement therapy, the administration of alglucosidase alfa, and Nexviazyme, all delivered via intravenous methods. An upswing in the number of individuals diagnosed with Pompe disease is set to bolster the market's trajectory. To illustrate, data from the European Medicines Agency Highlights in June 2022 indicated that, within Europe, approximately 224 individuals exhibited infantile-onset forms, 471 showed juvenile-onset forms, and an estimated 20,490 had adult-onset forms carrying mutations that could potentially manifest as Pompe disease, calculations derived from a peak birth incidence rate of 11.6 per 100,000. Consequently, the heightened occurrence of Pompe disease serves as a catalyst for the market's upward movement.
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What Long-Term Trends Will Define the Future of the Pompe Disease Market?
A significant development currently influencing the Pompe disease arena involves product introductions, a trend that is steadily increasing in prominence. Leading firms within this sector are actively pursuing novel developments as a core strategy for maintaining their competitive standing. To illustrate this, in September of 2023, Amicus Therapeutics, a biopharmaceutical entity headquartered in the United States, secured authorization from the U.S. Food and Drug Administration for its dual therapy consisting of Pombiliti (cipaglucosidase alfa-atga) alongside Opfolda (miglustat) in 65 mg capsule form. This specific treatment regimen is targeted towards adult individuals diagnosed with late-onset Pompe disease (LOPD) who meet a minimum weight criterion of 40 kilograms. The Pombiliti and Opfolda pairing is distinctive; Pombiliti functions as a recombinant human GAA enzyme (rhGAA) engineered specifically to enhance its penetration into muscle tissue, while Opfolda plays the complementary role of an enzyme stabilizer, ensuring the therapeutic enzyme remains stable while circulating in the bloodstream. This official endorsement by the FDA serves to highlight the crucial role that ongoing progress in both scientific research and medical application plays in this field.
Which Segments in the Pompe Disease Market Offer the Most Profit Potential?
The pompe diseasemarket covered in this report is segmented -
1) By Type: Classic Infantile Form, Non-Classic Infantile Form, Late-Onset Form, Other Types
2) By Diagnosis: Blood Test, Genetic Test, Prenatal Test, Other Diagnosis
3) By Route Of Administration: Oral, Parenteral, Other routes Of administrations
4) By Therapy Type: Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Gene Therapy, Other Therapies
5) By End Users: Hospitals Or Clinics, Pharmaceuticals, Researchers, Other End-Users
Subsegments:
1) By Classic Infantile Form: Early-Onset Classic Pompe Disease; Severe Classic Pompe Disease
2) By Non-Classic Infantile Form: Mild Non-Classic Infantile Pompe Disease; Variants of Non-Classic Infantile Form
3) By Late-Onset Form: Adult-Onset Pompe Disease; Juvenile-Onset Pompe Disease
4) By Other Types: Asymptomatic Carriers; Other Rare Variants of Pompe Disease
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Which Firms Dominate the Pompe Disease Market by Market Share and Revenue in 2025?
Major companies operating in the pompe disease market include F. Hoffmann-La Roche AG, Sanofi S.A., Amicus Therapeutics Inc., Sangamo BioSciences Inc., Asklepios BioPharmaceutical Inc., Audentes Therapeutics Inc., Genethon, Maze Therapeutics Inc., Avidity Biosciences Inc., EpiVax Inc., M6P Therapeutics, Oxyrane UK Limited, Valerion Therapeutics LLC, Abeona Therapeutics Inc., BioMarin Pharmaceutical, Genzyme Corporation, Novartis AG, Ultragenyx Pharmaceutical, Aro Biotherapeutics Company, Akashi Therapeutics, Protalix BioTherapeutics, AVROBIO Inc., Catabasis Pharmaceuticals
Which Regions Offer the Highest Growth Potential in the Pompe Disease Market?
North America was the largest region in the pompe disease market in 2024. The regions covered in the pompe disease market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
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