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Pompe Disease Market (Therapy Type - Enzyme Replacement Therapy, and Substrate Reduction Therapy; Molecule Type - Biologics, and Small Molecules; Dosage Form - Solid, and Liquid): Global Industry Analysis, Trends, Size, Share and Forecasts to 2028

Pompe Disease Market (Therapy Type - Enzyme Replacement Therapy, and Substrate Reduction Therapy; Molecule Type - Biologics, and Small Molecules; Dosage Form - Solid, and Liquid): Global Industry Analysis, Trends, Size, Share and Forecasts to 2028

Report Code: HC0906 Category: Healthcare & Medical Devices Published: July, 2022

A recent report published by Infinium Global Research on pompe disease market provides in-depth analysis of segments and sub-segments in the global as well as regional pompe disease market. The study also highlights the impact of drivers, restraints, and macro indicators on the global and regional pompe disease market over the short term as well as long term. The report is a comprehensive presentation of trends, forecast and dollar values of the global pompe disease market. According to the report, the global pompe disease market is projected to grow at a CAGR of over 4% over the forecast period of 2022-2028.

 

Market Insight

The global pompe disease market was valued at about USD 730 Million in 2022 and is expected to reach nearly USD 950 Million in 2028, with a CAGR of over 4% during the forecast period. Pompe disease is an autosomal recessive metabolic disorder that damages muscle and nerve cells throughout the body. It is caused by an accumulation of glycogen in the lysosome due to a deficiency of the lysosomal acid alpha-glucosidase enzyme. These diseases approximately affect one individual in 40,000 in the U.S, however, the incidence varies among different ethnic groups. Pompe disease can be treated in symptomatic, supportive, and disease-specific ways. Increasing developments in gene therapy and enzyme replacement therapies are the primary factors driving the growth of the pompe disease market. Moreover, inflated costs associated with pompe disease therapy could hinder the market growth opportunities.

 


Pompe Disease Market


The growth of the market is basically attributed to the launch of novel drugs and therefore the continued uptake of long-term therapies. Pompe disease is a rare disease classified as early infantile or late-onset majorly affecting musculus, heart defects, and breathing problems. As per the study by the National Institute of Health in April 2022, the disease affects approximately 1 in 40,000 patients. Moreover, consistent with the study titled "Carrier frequency and predicted genetic prevalence of pompe disease supported a general population database" genetic science and Metabolism report in June 2021, Pathogenic or likely pathogenic variants (PPVs) were found in higher proportions in East Asian and African patients with classic infantile-onset pompe disease. Within the general population, total carrier frequency (CF) and predicted genetic prevalence (PGP) was 1.3 % (1 in 77) and 1:23,232, respectively. Thus, the rising prevalence of pompe disease is predicted to fuel the market growth. The launching of novel drugs globally will propel the demand for treatment of this disorder and further provide lucrative growth opportunities for the market. However, the high cost of the treatment will restrain the expansion of the market during the forecast period.

 

The COVID-19 epidemic had an inconsequential effect on the pompe disease market during the forecast period. The outbreak of the COVID-19 epidemic has affected the production and logistics of medical products utilized for pompe disease patients. As per the study titled "COVID-19 infection in patients with late-onset pompe disease" published within the Muscles and Nerve in March 2022, in some pompe disease patients, COVID-19 infection can have a benign outcome. On Moreover hand, individuals with Pompe disease are still at high risk. Thus, COVID-19 significantly impacted the pompe disease market. Due to these factors, the expansion of the market was restrained during the covid-19 pandemic.

 

The global pompe disease market is segmented into the Asia Pacific, North America, Europe, the Middle East & Africa, and South America. Globally, North America holds the largest market share, of the market. However, product launches by the key market players with reference to the segment are anticipated to drive the market over the forecast period. As an example, In August 2021, The U.S. Food and Drug Administration (FDA) approved a replacement enzyme replacement therapy, avalglucosidase alfa, for patients aged 1 year and older with late-onset pompe disease. The European region is anticipated to be the second-largest revenue-making region during the forecast period. Experimental drugs prepared for pompe disease are gaining an increasing number of approvals within the region. For example, the Marketing Authorization Applications for AT-GAA, an investigational two-component medication introduced by Amicus Therapeutics for the treatment of Pompe disease, are verified by the ECU Medicines Agency (EMA). This will enhance the market growth within the European region. However, the Asia Pacific is anticipated to be the fastest growing region in pompe disease market during the forecast period. The expansion of the region is followed by the expansion in India and China because of the increased awareness of pompe disease and rising genetic prevalence within the countries, which have resulted in the continuous growth of the market.

 

Segment Covered

The report on the global pompe disease market covers segments such as therapy type, molecule type, and dosage form. On the basis of therapy type, the sub-markets include enzyme replacement therapy, and substrate reduction therapy. On the basis of molecule type, the sub-markets include biologics, and small molecules. On the basis of dosage form, the sub-markets include solid, and liquid.

 

Companies Profiled:

The report provides profiles of the companies in the market such as Amicus Therapeutics, BioMarin Pharmaceuticals, Genzyme, Audentes Therapeutics, EPIVAX, OXYRANE, Valerion Therapeutics, Sangamo Biosciences, Sanofi, and AVROBIO, Inc.

 

Report Highlights:

The report provides deep insights into the demand forecasts, market trends, and micro and macro indicators. In addition, this report provides insights into the factors that are driving and restraining the growth in this market. Moreover, The IGR-Growth Matrix analysis given in the report brings an insight into the investment areas that existing or new market players can consider. The report provides insights into the market using analytical tools such as Porter's five forces analysis and DRO analysis of pompe disease market. Moreover, the study highlights current market trends and provides forecast from 2022-2028. We also have highlighted future trends in the market that will affect the demand during the forecast period. Moreover, the competitive analysis given in each regional market brings an insight into the market share of the leading players.


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