Pompe Disease Market Outlook Report 2024-2033 | Size, Trends, Analysis, Insights, Overview

The pompe disease market size has grown steadily in recent years. It will grow from $1.66 billion in 2023 to $1.73 billion in 2024 at a compound annual growth rate (CAGR) of 4.2%. The growth in the historic period can be attributed to increased awareness and diagnosis, orphan drug designation and incentives, global collaborations and research initiatives, patient advocacy and support groups..

The pompe disease market size is expected to see strong growth in the next few years. It will grow to $2.13 billion in 2028 at a compound annual growth rate (CAGR) of 5.3%. The growth in the forecast period can be attributed to continued research and development, expansion of newborn screening programs, advancements in gene therapy, increasing global investment in rare diseases, patient-centric drug development, expansion of clinical trials and research initiatives, market emphasis on personalized medicine approaches. Major trends in the forecast period include product innovation, technological advancements, implementation of patient support programs, growing adoption of telemedicine for patient management, collaborations for expanded access programs..

Market Overview -

Pompe disease is a genetic condition in which a complex sugar called glycogen accumulates in the body's cells. The situation is caused by a lack of an enzyme known as acid alfa glucosidase (GAA), which breaks down complex carbohydrates in the body.

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Rising Prevalence Of Pompe Disease Fuels Market Expansion
The increase in the prevalence of pompe disease is expected to propel the growth of the pompe disease market. Pompe disease, also known as glycogen storage disease type II or acid maltase deficiency, is a rare genetic disorder characterized by the build-up of glycogen (a complex sugar molecule) in various body tissues, particularly in muscles and organs. Treatments for pompe disease include protein replacement therapy, alglucosidase alfa, and Nexviazyme for intravenous infusion. A rise in pompe disease cases will contribute to the growth of the pompe disease market. For instance, in June 2022, according to the European Medicines Agency Highlights, a Europe-based intergovernmental organization, there are roughly 224 infantile-onset, 471 juvenile-onset, and 20,490 adult-onset persons in Europe with mutations that potentially cause Pompe disease based on the maximum birth incidence of 11.6 per 100,000. Therefore, the increase in the prevalence of pompe disease is driving the growth of the pompe disease market.

Competitive Landscape -

Major companies operating in the pompe disease market report are F. Hoffmann-La Roche AG, Bayer AG, Novartis AG, Sanofi S.A., Thermo Fisher Scientific Inc., Fresenius Kabi AG, Danaher Corporation, Becton, Dickinson and Company, Astellas Pharma Inc., BioMérieux SA, BioMarin Pharmaceutical Inc., Nova Biomedical Corporation, Instrumentation Laboratory Company, Amicus Therapeutics Inc., Sekisui Diagnostics LLC, Spark Therapeutics Inc., Sangamo BioSciences Inc., Asklepios BioPharmaceutical Inc., Avrobio Inc., EKF Diagnostics Holdings plc, Trinity Biotech plc, Chembio Diagnostics Inc., Audentes Therapeutics Inc., Rougine Darou, Genethon, Maze Therapeutics Inc., Avidity Biosciences Inc., EpiVax Inc., M6P Therapeutics, Oxyrane UK Limited, Valerion Therapeutics LLC

Innovative Therapies Transforming Pompe Disease Market Dynamics
Product innovations are a key trend gaining popularity in the pompe disease market. Major companies operating in the pompe disease market are adopting innovations to sustain their position in the market. For instance, in September 2023, Amicus Therapeutics, a US-based biopharmaceutical company, received approval from the U.S. Food and Drug Administration (FDA) for Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65 mg capsules. This two-component therapy is designed for adults with late-onset Pompe disease (LOPD) weighing =40 kg. Pombiliti and Opfolda are a unique combination, with Pombiliti being a recombinant human GAA enzyme (rhGAA) designed for increased uptake into muscle cells and Opfolda serving as an enzyme stabilizer to stabilize the enzyme in the blood. This FDA approval underscores the significance of advancements in science and medicine.

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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 / Clinics, Pharmaceuticals, Researchers, Other End-Users

Key highlights covered in the report -

1. Detailed market size forecast and historical data analysis
2. Key drivers influencing market growth
3. Identification of upcoming trends and potential opportunities in the market
4. Analysis of major players strategies, to understand competitive dynamics and market positioning
5. Evaluation of regional dynamics

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