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Pompe Disease Treatment Market Size, Share and Global Trend By Treatment (Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Chaperone-Advanced Replacement Therapy (CART), Others), By Rout of Administration (Oral, Intravenous, Others), By Distribution channel (Hospital & Clinics Pharmacies, Retail Pharmacies, Online Pharmacies, Others) and Regional Forecast, 2024-2032

Region : Global | Report ID: FBI101008 | Status : Ongoing



Pompe disease is an inherited disorder caused due to the accumulation of a complex sugar called glycogen in the body's cells. Pompe disease is an autosomal recessive glycogen storage disorder caused due to the deficiency of lysosomal enzyme acid α-glucosidase (GAA). The accumulation of glycogen in specific organs and tissues, especially muscles, impairs their ability to function normally. Pompe disease has been classified into three different types based on their time of onset and its severity.

Approximately 20% of patients with Pompe disease have the infantile or early-onset form of the disease, and the more common form of the disease is known as juvenile or adult-onset form, reported in 80% of patients suffering from Pompe disease. The condition primarily affects cardiac and skeletal muscle and manifests as cardiomyopathy, progressive respiratory distress, and skeletal muscles atrophy.

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Increasing prevalence of Pompe disease and developing healthcare infrastructure especially in emerging countries are some of the major factor driving the growth of the global Pompe disease treatment market. According to the National Organization for Rare Disorders, Inc., the incidence is generally placed at approximately 1 in 40,000 births in the United States.

Increasing investment in the research and development in the gene therapy and enzyme replacement therapies are some of the major factors driving the growth of the Pompe disease treatment market. Increased adoption and demand of the Enzyme Replacement Therapy (ERT) is one of the major factor anticipated to drive the growth of the global Pompe disease treatment market.

The poor response of skeletal muscles to alglucosidase alpha treatment and the inadequate reimbursement policies are some of the factors restraining the growth of the global Pompe disease treatment market.

Key Players Covered

Some of the major companies that are present in the global Pompe disease treatment market is Genzyme Corporation, Amicus Therapeutics, Inc., Valerion Therapeutics, Audentes Therapeutics, EpiVax, Inc., Oxyrane, Sangamo Therapeutics. and others. 




By Treatment

· Enzyme Replacement Therapy (ERT)

· Substrate Reduction Therapy (SRT)

· Chaperone-Advanced Replacement Therapy (CART)

· Others

By Route of Administration

· Oral

· Intravenous

· Others

By Distribution channel

· Hospital & Clinics Pharmacies

· Retail Pharmacies

· Online Pharmacies

· Others

By Geography

· North America (the USA and Canada)

· Europe (UK, Germany, France, Italy, Spain, Scandinavia and Rest of Europe)

· Asia Pacific (Japan, China, India, Australia, Southeast Asia and Rest of Asia Pacific)

· Latin America (Brazil, Mexico and Rest of Latin America)

· Middle East & Africa (South Africa, GCC and Rest of the Middle East & Africa)


In 2018, among treatment, enzyme replacement therapy (ERT) dominated the global Pompe disease treatment market due to the high demand and the therapy being the only long-term therapies available for the treatment of the Pompe disease currently.

Key Insights

  • Prevalence of Pompe disease by key countries

  • Overview of development in enzyme replacement therapy (ERT) & gene therapy.

  • New Product launches, Key Market Players

  • Pipeline Analysis

  • Recent Industry Developments Such as Partnerships, Mergers, and Acquisitions

Regional Analysis

North America dominated the global Pompe disease treatment market in 2018. Increasing prevalence of the Pompe disease in the region and increasing healthcare expenditure are some of the major factors driving the growth of the Pompe disease treatment market. According to US National Library of Medicine National Institutes of Health, the prevalence of Pompe disease is approximately 1 in 28,000 in the United States. The Asia Pacific is anticipated to grow at significant CAGR due to increasing awareness among patients regarding rare disorders and improved healthcare facilities in Asian countries.

Key Industry Developments

  • In February 2019, Amicus Therapeutics, Inc., received U.S. Food and Drug Administration (FDA) approval for the Breakthrough Therapy Designation (“BTD”) to AT-GAA for the treatment of late-onset Pompe disease. AT-GAA is a novel treatment model consisting of ATB200, a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, co-administered with AT2221, a pharmacological chaperone.

  • In April 2018, Duke University and Duke University Health System announced the beginning of the clinical trials for Pompe Disease gene therapy. Researchers are currently screening adults with late-onset Pompe disease as phase I clinical trial to test the safety of the treatment in 20 people with the condition

  • Ongoing
  • 2023
  • 2019-2022

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