Pompe Disease Treatment Market Trends

Global Pompe Disease Treatment Market Overview

Pompe Disease Treatment Market Size was valued at USD 1.14 Billion in 2023. The pompe disease treatment market industry is projected to grow from USD 1.183 Billion in 2024 to USD 1.538 Billion by 2032, exhibiting a compound annual growth rate (CAGR) of 3.33% during the forecast period (2024 - 2032). The market for pompe disease treatments is expanding as a result of market drivers including rising pompe disease prevalence and increasing healthcare infrastructure, particularly in emerging nations. Increasing funding for research and development in gene therapy and enzyme replacement medicines is another important element boosting the market for treating pompe illness.

Source: Secondary Research, Primary Research, MRFR Database and Analyst Review

May 2024: Shionogi has finalized an exclusive worldwide license agreement for Maze Therapeutics's MZE001, an oral inhibitor of glycogen synthase 1 (GYS1) used to treat Pompe disease. Shionogi has been given exclusive rights to MZE001 and its related programs, as well as the intellectual property associated with them, worldwide. Shionogi is obligated to provide Maze with an advance payment of $150 million. Maze will be entitled to receive milestone payments based on the advancement of the development, regulatory authorizations, and commercial triumph. In addition, the company will receive graduated royalties based on the future net sales of the therapeutic candidate. MZE001 functions by inhibiting the buildup of pathogenic glycogen. It impedes the activity of GYS1, an enzyme that plays a crucial role in the process of glycogen production. This inhibition decreases the buildup of glycogen in muscles, which is a crucial element in Pompe illness.

In February 2024, Sanofi announced favorable outcomes for its enzyme replacement medication, avalglucosidase alfa, in various patient groups affected by Pompe disease. Pompe disease, a rare genetic disorder, affects around one in every 40,000 individuals in the UK. This condition is caused by insufficient amounts of the enzyme acid alpha-glucosidase. This leads to the accumulation of glycogen in muscle cells throughout the body, which can cause possibly irreparable damage to the heart and skeletal muscles. Pompe disease can manifest as either infantile-onset Pompe disease (IOPD), the most severe type of the disease that rapidly appears in infancy, or late-onset Pompe disease (LOPD), which gradually causes muscle degeneration over time. Untreated IOPD can result in cardiac difficulties and mortality within the initial year of life, whereas individuals with LOPD may necessitate mechanical ventilation for respiratory support or a wheelchair for mobility assistance as the condition advances. The findings from the mid-stage Mini-COMET study long-term extension, presented at this year's WORLDSymposium, indicates that avalglucosidase alfa led to significant improvements in ptosis (drooping eyelid) in paediatric patients with IOPD over a period of almost three years.

Pompe Disease Treatment Market Trends

• Incidence of genetic defects is driving the market growth

The incidence of genetic defects among people has significantly increased, as has the number of people with pompe disease. One of the main factors boosting the market's growth is this, along with the rising prevalence of pompe disease in infants and parents' increased concerns about the health of their newborns. Early infantile or late-onset pompe disease is an uncommon condition that mostly affects the skeletal muscle, causes cardiac abnormalities, and causes breathing issues. For instance, 1 in 40,000 Americans has pompe disease, according to a National Institutes of Health report dated April 2022. Additionally, the research study from June 2021 discovered that patients with the classic infantile-onset Pompe illness from East Asia and Africa had greater proportions of pathogenic or potentially pathogenic variations (PLPVs). The same source also said that the total carrier frequency (CF) and anticipated genetic prevalence (pGP) in the overall population were 1.3 percent (1 in 77) and 1:23,232, respectively. Therefore, it is projected that during the course of the forecast period, the increased prevalence of pompe disease will spur market CAGR.

Acid alpha-glucosidase is lacking in the body as a result of the genetic mutation, which reduces muscle strength and causes weariness. People are increasingly turning to therapies that implant external enzymes and replace missing ones as a result of this shortage. By supplying the missing enzymes, these treatments enable the patient's body to function properly. As a result, in recent years, the demand for such therapies has increased. In order to meet this need, hospitals and research facilities are always coming up with innovative ways to treat this enzyme deficit, which is further boosting the growth of the worldwide pompe disease treatment market.

The introduction of new medications is projected to increase demand for the management of pompe disease, which might further fuel market expansion. Furthermore, as pompe disease is primarily fatal if not treated early on, boosting R&D spending internationally will encourage study on innovative medicines and the treatment of this condition. For instance, using technology created by Genethon, the startup Spark Therapeutics began a clinical trial for late-onset pompe disease in February 2021. Additionally, given that this therapy is widely utilized to treat this ailment, rising advancement in gene therapy and enzyme replacement therapies may potentially generate a lucrative opportunity for the pompe disease treatment market over the projection period. Therefore, it is projected that throughout the course of the forecast period, increased product releases and research and development related to pompe disease will fuel pompe disease treatment market revenue.

Pompe Disease Treatment Market Segment Insights

Pompe Disease Treatment Type Insights

The pompe disease treatment market segmentation, based on type includes Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease and Non-Classic infantile-onset Pompe Disease. The late-onset pompe disease segment dominated the market. LOPD is characterized by a progressive respiratory insufficiency and muscle weakness that can start at any age older than 12 months and develop to early disability, assisted breathing, and death. Before muscular weakness, frequent early symptoms that can appear anywhere from childhood through late adulthood include myalgia, activity intolerance, and tiredness. Clinical approvals for alglucosidase alfa have increased as a result of its effectiveness as an ERT on motor and respiratory endpoints in LOPD patients.

Pompe Disease Treatment Therapy Insights

The pompe disease treatment market segmentation, based on therapy, includes Gene Therapy and Enzyme Replacement Therapy. The enzyme replacement therapy category generated the most income. Patients who experience chronic illnesses brought on by an enzyme deficit or dysfunction can receive ERT, or enzyme replacement therapy, in which replacement enzymes are given to them. The most popular form of ERT is intravenous infusions, in which the replacement enzyme is administered directly into the bloodstream using a carefully regulated fluid flow. Additionally, the effectiveness of enzyme replacement therapy (ERT), the rise in Pompe disease patients, and increased funding for research into rare diseases are the primary drivers for the market.

Figure 1: Pompe Disease Treatment Market, by Therapy, 2023 & 2032 (USD Billion) 

Source: Secondary Research, Primary Research, MRFR Database and Analyst Review

Pompe Disease Treatment End-User Insights

 The pompe disease treatment market segmentation, based on end-user, includes Hospitals and Clinics and Research and Academic Institutes. The hospitals and clinics category generated the most income. With the aim of sending patients with vague symptoms to specialist centers as soon as possible, numerous hospitals are creating fresh and enhanced methods to aid in the early detection of pompe disease. Patients would be able to receive timely access to efficient disease management since they would be able to receive an accurate assessment and diagnosis.

Pompe Disease Treatment Regional Insights

By region, the study provides the market insights into North America, Europe, Asia-Pacific and Rest of the World. The North American pompe disease treatment market area will dominate this market due to the presence of established payers and an expansion in the region's population of people with pompe disease. This expansion can be ascribed to the expansion of R&D projects and government assistance for the expansion of the healthcare industry. The government has taken steps to encourage healthcare spending, and people' increasing knowledge of uncommon ailments in this area is one of the main drivers of the market for pompe disease treatments.

Further, the major countries studied in the market report are The US, Canada, German, France, the UK, Italy, Spain, China, Japan, India, Australia, South Korea, and Brazil.

Figure 2: POMPE DISEASE TREATMENT MARKET SHARE BY REGION 2023 (USD Billion) 

Source: Secondary Research, Primary Research, MRFR Database and Analyst Review

Europe pompe disease treatment market accounts for the second-largest market share. In recent years, a growing number of experimental medications developed for the treatment of pompe disease have been approved. Additional factors that are anticipated to have a substantial impact on the region's market revenue include increased financing and partnerships with big corporations to undertake clinical research on the efficacy of novel therapeutic alternatives for the treatment of Pompe disease. Further, the German pompe disease treatment market held the largest market share, and the UK pompe disease treatment market was the fastest growing market in the European region

The Asia-Pacific Pompe disease treatment Market is expected to grow at the fastest CAGR from 2023 to 2032. Due to rising genetic prevalence and higher awareness of pompe disease in developing nations, the pompe disease market is expanding in this region, particularly in India, Japan, and China. This sector is expected to continue growing. Additionally, the market is expanding as a result of greater government and pharmaceutical organization initiatives to raise awareness as well as the presence of generic producers. Moreover, China’s pompe disease treatment market held the largest market share, and the Indian pompe disease treatment market was the fastest growing market in the Asia-Pacific region.

Pompe Disease Treatment Key Market Players & Competitive Insights

Leading market players are investing heavily in research and development in order to expand their product lines, which will help the pompe disease treatment market, grow even more. Market participants are also undertaking a variety of strategic activities to expand their footprint, with important market developments including new product launches, contractual agreements, mergers and acquisitions, higher investments, and collaboration with other organizations. To expand and survive in a more competitive and rising market climate, pompe disease treatment industry must offer cost-effective items.

Manufacturing locally to minimize operational costs is one of the key business tactics used by manufacturers in the pompe disease treatment industry to benefit clients and increase the market sector. In recent years, the pompe disease treatment industry has offered some of the most significant advantages to market. Major players in the pompe disease treatment market attempting to increase market demand by investing in research and development operations include Amicus Therapeutics, Inc (US), Sanofi (France), Audentes Therapeutics (US), Oxyrane (UK), Valerion Therapeutics (US), AVROBIO, Inc. (US), and CENTOGENE AG (UK).

The development of biotherapies for rare genetic illnesses is the primary objective of the newly established, science-driven firm Valerion Therapeutics. In order to penetrate the cytoplasm of afflicted tissues, which was previously inaccessible for protein-based therapies, Valerion uses a specialized cell-penetrating antibody. Its flexible platform can be coupled to proteins, small compounds, and oligonucleotides, offering a fresh approach to treat diseases that were previously untreatable.

Amicus Therapeutics Inc. (Amicus Therapeutics) is a biotechnology firm that specializes in the study, creation, and marketing of small molecule medications and pharmaceutical chaperones for oral administration for the treatment of uncommon and orphan illnesses. In February 2020, the safety, PK, effectiveness, PD, and immunogenicity of the ATB200/AT2221 medication in pediatric patients with pompe disease, aged 0 to 18 years old, are the subjects of a Phase III clinical trial investigation by Amicus Therapeutics.

Key Companies in the pompe disease treatment market include

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  Amicus Therapeutics, Inc (US)

  
  


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  Sanofi (France)

  
  


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  Audentes Therapeutics (US)

  
  


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  Oxyrane (UK)

  
  


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  Valerion Therapeutics (US)

  
  


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  AVROBIO, Inc. (US)

  
  


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  CENTOGENE AG (UK)

  
  

Pompe Disease Treatment Industry Developments

June 2022:For the long-term treatment of both infantile-onset and late-onset Pompe disease, a rare, degenerative, and crippling muscle illness, the European Commission approved the sale of Nexvia Dyme (alglucosidase alfa), an enzyme replacement therapy (ERT).

February 2022:MZE001, which intends to treat Pompe illness by lowering pathologic glycogen accumulation by inhibiting muscle glycogen synthase (GYS1), has received fresh preclinical data in favor of its development, according to Maze Therapeutics, a business that converts genetic insights into innovative precision medicines.

March 2021:In order to evaluate the effectiveness and safety of Alglucosidase Alfa treatment in Chinese patients with LOPD, Genzyme, a Sanofi Company, has been carrying out a Phase IV clinical research investigation.

Pompe Disease Treatment Market Segmentation

Pompe Disease Treatment Type Outlook (USD Billion, 2018-2032)

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  Late-Onset Pompe Disease

  
  


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  Classic Infantile-Onset Pompe Disease

  
  


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  Non-Classic infantile-onset Pompe Disease

  
  

Pompe Disease Treatment Therapy Outlook (USD Billion, 2018-2032)

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  Gene Therapy

  
  


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  Enzyme Replacement Therapy

  
  

Pompe Disease Treatment End-User Outlook (USD Billion, 2018-2032)

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  Hospitals and Clinics

  
  


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  Research and Academic Institutes

  
  

Pompe Disease Treatment Regional Outlook (USD Billion, 2018-2032)

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  Asia-Pacific

  
  
  
  
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    China

    
    
  
  
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    Japan

    
    
  
  
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    India

    
    
  
  
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    Australia

    
    
  
  
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    South Korea

    
    
  
  
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    Australia

    
    
  
  
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    Rest of Asia-Pacific

    
    
  
  
  
  


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  Rest of the World

  
  
  
  
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    Middle East

    
    
  
  
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    Africa

    
    
  
  
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    Latin America