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How has the pompe disease market size evolved in recent years?

There has been a consistent expansion in the market size for Pompe disease in recent years. The market is anticipated to rise from $1.74 billion in 2024 to around $1.8 billion in 2025, achieving a compound annual growth rate (CAGR) of 3.6%. The historic growth is a result of heightened awareness and diagnosis, incentives and designation of orphan drugs, international collaborations, research initiatives, patient advocacy, and the existence of support groups.

What are the predictions for the pompe disease market size in the coming years?

The market for pompe disease is anticipated to exhibit robust expansion in the upcoming years, reaching $2.32 billion by 2029 with a CAGR of 6.5%. The projected growth over the forecast period primarily stems from sustained R&D efforts, broadening of newborn screening initiatives, improvements in gene therapy, an increase in worldwide investment in rare diseases, and a focus on patient-driven drug invention. Extensive clinical trials and research projects, as well as a market concentration on individualized medicine approaches, also play a part. Key trends during the forecast period are likely to involve product innovation, technological progression, the introduction of patient support programs, rising utilization of telemedicine for patient supervision, and partnerships aimed at widening access programs.

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What key factors are fueling the growth of the pompe disease market?

The pompe disease market is anticipated to expand due to the rising cases of pompe disease. This rare genetic disorder, also known as glycogen storage disease type II or acid maltase deficiency, causes an accumulation of glycogen (a complex sugar molecule) in various tissues of the body, particularly muscles and organs. The treatment options for this disease include protein replacement therapy, alglucosidase alfa, and Nexviazyme for intravenous infusion. The augmentation in the occurrence of the disease will boost the market development. For example, the European Medicines Agency Highlights reported in June 2022 that in Europe, an intergovernmental institution, around 224 infantile-onset, 471 juvenile-onset, and 20,490 adult-onset individuals carry mutations potentially leading to Pompe disease, with the highest birth incidence standing at 11.6 per 100,000. Hence, the growth in the prevalence of pompe disease plays a crucial role in propelling the pompe disease market.

How is the global pompe disease market divided into key segments?

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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Who are the key firms paving the way for growth 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

Which trends are expected to transform the pompe disease market?

In the pompe disease market, the rising trend of product innovations is evident. Major firms in this market are embracing these innovations to maintain their standing. For example, Amicus Therapeutics, a biopharmaceutical firm based in the United States, was granted approval for Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65 mg capsules by the U.S. Food and Drug Administration (FDA) in September 2023. These therapies were developed for adults suffering from late-onset Pompe disease (LOPD) who weigh ≥40 kg. The unique blend of Pombiliti, a recombinant human GAA enzyme (rhGAA) intended to enhance uptake into muscle cells, and Opfolda, an enzyme stabilizer, enhances the stability of the enzyme in the bloodstream. This approval from the FDA highlights the importance of progress in the fields of science and medicine.

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What regions are contributing significantly to the growth of 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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