What Has Been the Growth Trajectory for the Pompe Disease Market in Recent Years?
The pompe disease market has seen considerable growth due to a variety of factors.
•In recent years, the size of the Pompe disease market has been on a consistent rise. The market is expected to increase from $1.74 billion in 2024 to $1.8 billion in 2025, indicating a compound annual growth rate (CAGR) of 3.6%.
Such growth during the historic period has been driven by factors like heightened awareness and diagnosis, designations and incentives for orphan drugs, collaborations and research initiatives across the globe, patient advocacy, as well as support groups.
How Does the Forecast Look for the Pompe Disease Market?
The pompe disease market is expected to maintain its strong growth trajectory in upcoming years.
• Anticipated to see robust growth in the coming years, the pompe disease market is predicted to expand to $2.32 billion in 2029, with a compound annual growth rate (CAGR) of 6.5%.
This growth over the forecasting period can be accredited to constant research and development efforts, the broadening of newborn screening programs, progress in gene therapy, a surge in global investment for rare diseases, a patient-focused approach to drug development, growth in clinical trials and research initiatives, and a market focus on personalized medicine methodologies. Key trends during this forecast period consist of product innovation, advancements in technology, establishment of patient aid programs, increased incorporation of telemedicine for patient care, and collaborative efforts for broadened access programs.
What Are the Critical Driver Fuelling the Pompe Disease Market's Growth?
The surge in the occurrence of pompe disease is slated to stimulate the expansion of the pompe disease market. This disease, also referred to as acid maltase deficiency or glycogen storage disease type II, is a unique genetic disorder where a complex sugar molecule, glycogen, amasses in various body tissues, predominantly in muscles and organs. The treatment options for this condition include protein replacement therapy, alglucosidase alfa, and Nexviazyme for intravenous infusion. The ascent in the cases of pompe disease is poised to contribute to the enhancement of the pompe disease market. For instance, data from the European Medicines Agency Highlights, a Europe-based intergovernmental organization, suggests that in June 2022, there were around 224 cases of infantile-onset, 471 cases of juvenile-onset, and 20,490 cases of adult-onset in Europe with potential Pompe disease causing mutations based on the maximum birth incidence rate of 11.6 per 100,000. Hence, the upswing in the incidence of pompe disease is responsible for propelling the growth of the pompe disease market.
How Is the Pompe Disease Market segmented?
The pompe disease market 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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What New Trends Are Influencing the Pompe Disease Market?
Introducing innovative products is a trend that is gaining traction in the pompe disease market. The major players in this market are adopting new and innovative approaches to maintain their market standing. For example, in September 2023, the U.S. Food and Drug Administration (FDA) approved two drugs - Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65 mg capsules for Amicus Therapeutics, a biopharmaceutical firm based in the US. These drugs, intended for adults suffering from late-onset Pompe disease (LOPD) who weigh =40 kg, represent a unique combination. While Pombiliti is a recombinant human GAA enzyme (rhGAA) created to increase uptake into muscular cells, Opfolda acts as an enzyme stabilizer, maintaining the enzyme’s stability in the blood. This approval from the FDA highlights the ongoing importance of scientific and medical advancements.
Who Are the Key Players in the Pompe Disease Market?
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
What Is The Most Dominant Region 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.
