Pompe Disease Treatment Market Research Report - Global Forecast till 2030

Pompe Disease Treatment Market: Information by Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, Non-Classic infantile-onset Pompe Disease), Therapy (Enzyme Replacement Therapy, Gene Therapy, Others), End-User (Hospitals and Clinics; Research and Academic Institutes) and Region (Americas, Europe, Asia-Pacific, and the Middle East & Africa) - Forecast till 2030

ID: MRFR/HC/6761-CR | January 2020 | Region: Global | 123 Pages         

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Pompe Disease Treatment Market Overview

The pompe disease treatment market is anticipated to reach USD 1,409.09 Million by 2030 at 3.80% CAGR during the forecast period 2022-2030. Pompe disease is a rare and fatal neurological disorder that causes the degeneration of the brain cells. This leads to tremors, seizures, and cognitive impairment. The Pompe disease treatment market has been growing steadily over the past few years. The prevalence of Pompe disease in the U.S is estimated at about 1 in 200,000 people with an annual incidence rate of about 1 case per 800,000 people. Pompe disease affects many different parts of individuals’ lives including their professional lives as it negatively impacts their career prospects.

Pompe Disease Treatment Market consists of both direct-to-consumer (DTC) and healthcare providers. DTC market includes various treatment options like stem cell transplant, bone marrow transplantation, chemotherapy, immunotherapy etc. Healthcare providers include academic medical centres and private practices.

COVID-19 Analysis of Pompe Disease Treatment Market

The pandemic caused by COVID-19 has affected the supply chain and the production of medical products for Pompe disease. The market may face disruption due to a lack of attendance of human labour at work and in factories. However, there is a silver lining. Just like any other market, advanced technology has taken place instead of human labour. Though it may need human interventions from time to time, it is indeed scarce.

The government of various nations are running vaccine drives. The initial months of the COVID-19 pandemic saw a decline in production of the market. However, the Pompe Disease Treatment Market has paced up production.

Pompe Disease Treatment Market Share, by Type, 2018 (%)  Pompe Disease Treatment Market

Source: MRFR Analysis

Market Dynamics


The Pompe Disease Treatment Market is driven by several factors such as increasing awareness, legislation, reimbursement policies of health insurance companies, and technological advancements. Pompe disease treatment market has been on the rise globally due to increasing awareness about the disease as well as its potential impact on society. The success of treatments is also strongly influenced by changes in legislation such as laws concerning reimbursement for Pompe Disease Treatment by health insurance companies.

The global healthcare industry continues to adopt new technologies every day which would positively influence Pompe Disease Treatment market size in future years. The treatment market for Pompe disease is growing due to the increase in the prevalence of the disease. The drivers for this market are increasing incidence rates, the presence of Pompe disease in developing countries, and the lack of effective treatments for this disease.


While the Pompe disease treatment market is expected to grow at a high rate in the coming years, it faces several restraints such as lack of well-defined efficacy, high cost, and drug regulatory issues.

Pompe disease is not curable but many therapies can help manage its symptoms. The main therapy includes enzyme replacement therapy or ERET which provide an alternative to the current therapies for the treatment of Pompe disease.


In the last few years, awareness about Pompe disease has been increasing in the global audience. This is because of the increasing number of people diagnosed with this hereditary disorder.

Pompe disease is a disorder that affects a person’s muscle and brain function. Patients with this disorder experience muscle spasms and eventually lose control over their bodily functions resulting in death. However, there are many treatment options available for patients to help manage the symptoms of the condition. The market size for these treatments is expected to grow USD 4 billion by 2025.

The growing prevalence of Pompe disease provides opportunities for treatment providers who have developed new products and services focused on improving patient outcomes through medication, medical devices, pharmaceuticals, surgical procedures, rehabilitation therapies and other interventions.


The treatment for this condition is expensive, and only about 1/3 of patients who receive it survive beyond five years. There’s a high degree of unmet medical need for Pompe disease treatment, which means that the market for this type of therapy is ripe for disruption.

Many challenges are facing the Pompe Disease Treatment Market, but one of the most significant ones is low adoption rates because people with this condition are not able to afford treatment.

Value Chain Analysis

As per the value chain analysis of the Pompe Disease Treatment Market, there are different stakeholders involved in the market such as pharmaceutical companies, healthcare providers, medical professionals and patients. The key players in this niche are Pfizer Inc., Hospira Inc., AstraZeneca PLC and Merck & Co., Inc.

Some companies are now developing new methods to treat the disease, such as the use of antibiotics combined with gene therapies. However, these methods are still not easy for people with low incomes to afford. Other diseases require treatment but there aren't many solutions available for them either.

The value chain can be divided into three parts: basic research, innovation, and commercialization. Basic research focuses on scientific discovery, innovation creates novel technologies, and commercialization creates marketable products ready for consumers. The value chain also includes healthcare providers.

Pompe Disease Treatment Market Share, by Region, 2018 (%)  Pompe Disease Treatment Market

Source: MRFR Analysis

Pompe Disease Treatment Market Segment Overview

Technology Types:

There are five types of Pompe Disease Treatment Market:

  • an oral medication

  • injection therapy

  • dietary interventions

  • gene therapy

  • bone grafting surgery


  • This increase in demand for the treatment has led to innovation and research about new methods of treating this condition such as physical therapy, surgery, and stem cell transplantation.

  • A new drug could change all of that. The drug is called Afrezza, and it could prove to be a game-changer for children with Pompe disease - if it can make it to market in time.

  • The market is expected to grow significantly due to the significant increase in demand for new treatments, availability of novel diagnostic tools, and awareness about the disease from various healthcare providers.


Regional Analysis

Pompe disease treatment market is expected to reach USD 1.5 billion by 2025 with the presence of global key players like Bayer AG, BMS, and CSL. The regional Pompe Disease Treatment market is analyzed through the use of a bottom-up approach. North America is projected to dominate the global Pompe Disease Treatment market in terms of revenue share by 2025.

The regional analysis of Pompe Disease Treatment Market report includes key insights on top markets including North America, Europe, Asia Pacific & the Rest of the World (RoW), The United States holds the largest share of the market while Italy has the highest revenue share among other countries in Europe. North America has been the largest regional market in 2015 and accounted for more than 50% of the overall global Pompe Disease Treatment Market share. With its high disposable income, North America is expected to maintain its dominance.

Competitive Landscape

With the increase in the number of cases of Pompe Disease, treatment and prevention have become a priority for both national and global health organizations.

The US National Institutes of Health (NIH) conducted a study to find out which is the best drug for Pompe Disease treatment. The study revealed that small molecule drugs like pegylated liposomal doxorubicin (DOXO) and pomalidomide may be more effective than other drugs like cilengitide and panobinostat.

However, the study also revealed that these drugs may not be affordable enough for most patients with Pompe Disease. Hence, there is a need to explore alternative therapies that can reduce the cost of treatment and prevention.

The Pompe Disease treatment market is growing exponentially. With this increase in demand for new treatments, the competition is fierce among the pharmaceutical industry players. The challenge for this company will be to stay ahead of its competitors.

  • Ilumya Therapeutics

  • Aralez Pharmaceuticals

  • Teva Pharmaceutical Industries

  • Roche Holding

Recent Developments

  • A new drug called Pexion is currently the only treatment option for Pompe disease. Pexion is a medication that works by slowing down the production of androgens and decreasing androgen and estrogen levels in the body.

  • With the recent advancements in medical technology, there are now various treatments available for Pompe diseases. Some of these treatments include drugs, gene therapy, stem-cell transplantation, bone marrow transplantation, gene editing etc.

  • One of the most promising treatments is gene editing. Recent research has shown that altering DNA to prevent the disease from happening in offspring, it increases the chances of success by 100%. However, it can only be done if both parents are carriers of the disease and only one parent has Pompe disease.

Report Overview

The Pompe Disease Treatment Market report traces different sectors of the market like drivers, opportunities, restraints and others. Pompe disease is rare and expensive to treat. It is also a non-curable disease. Developments in the treatments of the disease are still going on.

  • Historic Period: 2015-2019

  • Base Year: 2028

  • Forecast Period: 2021-2028


By Technology

This disease has an average life expectancy of three to five years. It can be fatal if left untreated. However, there is hope for this deadly disease. Pompe Disease Treatment uses gene therapy to stop the immune system from attacking neurons and stop the progression of the disease.

By Service

Pompe disease treatment includes hospitalization followed by intravenous injections of drugs which help patients to maintain cognitive function until their bodies can no longer produce enough enzymes to break down toxic metabolites.

Report Scope:

Report Attribute/Metric Details
  Market Size   USD 1,409.09 Million
  CAGR   3.80%
  Base Year   2021
  Forecast Period   2022-2030
  Historical Data   2020
  Forecast Units   Value (USD Million)
  Report Coverage   Revenue Forecast, Competitive Landscape, Growth Factors, and Trends
  Segments Covered   Type, Therapy, End-User and Region
  Geographies Covered   North America, Europe, Asia-Pacific, and Rest of the World (RoW)
  Key Vendors   Amicus Therapeutics, Inc (US) Sanofi (France), Audentes Therapeutics (US), Oxyrane (UK), Valerion Therapeutics (US), AVROBIO, Inc. (US), CENTOGENE AG (UK).
  Key Market Opportunities   The rising prevalence of Pompe disease is creating opportunities for manufacturers to find new solutions for the diagnosis and treatment
  Key Market Drivers

  • Increasing research & development initiatives
  • Government support for improving the healthcare sector

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    Frequently Asked Questions (FAQ) :

    Pompe disease treatment market was valued at close to USD 1,409.09 Million in 2030.

    Global market is expected to exhibit a strong 3.80% CAGR over the forecast period from 2022-2030.

    Increasing R&D initiatives by drug manufacturers are the major driver for the pompe disease treatment market.

    The Americas hold the dominant share in the global market, followed by Europe.

    Leading players in the market include Amicus Therapeutics, Valerion, and Sanofi, among others.