Pompe Disease Therapeutic Market Outlook 2020 – Global Analysis, Growth Rate, Modern Trend, Demographical Dynamics and Analysis of Key Strategies By 2027

SEATTLE, March 17, 2021, (PHARMIWEB) — Deficiency of enzyme acid named alpha-glucosidase (GAA) is a major cause of Pompe disease, an inherited Lysosomal Storage Disorder (LSD). Deficiency of GAA leads to accumulation of glycogen in different kinds of tissues, primarily into smooth muscles, skeletal muscles and cardiac muscle, which causes weakness in the muscles, respiratory problems, and may also affect the liver and heart. Pompe disease is inherited as an autosomal recessive genetic trait. Based on severity and the stage at which the disease occurs, pompe disease is classified into classic infantile-onset, which starts within a few months of birth, non-classic infantile-onset Pompe disease- usually appears by age 1, and late-onset type of Pompe disease that may shows the symptoms later in childhood, adolescence, or adulthood. The Pompe disease therapeutic market is expected to witness significant growth, owing to increasing expansion activities adopted by manufacturers in the market.

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Pompe Disease Therapeutic Market Taxonomy:

By Therapy Type:

• Gene Therapy
• Enzyme Replacement Therapy (ERT)
• Chaperone-Advanced Replacement Therapy (CHART)

By Administrational Route:

• Oral
• Parenteral

By Patient Type:

• Infants
• Adults

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Increasing government initiatives to fund and support treatment of Pompe disease is expected to boost the market growth

Several governments around the worlds are focused on supporting the development of innovative therapies for the diagnosis and treatment of rare diseases or conditions such as Pompe disease. For instance, in 2015, Orphan Product Grants Program funded 18 new grant awards from 92 grant applications and provided funding and support to around 67 ongoing clinical study projects related to rare disease. Moreover, governments in Sweden, the Netherlands, and South Korea are focused on approving guidelines for the reimbursement of individuals undergoing enzyme replacement therapy (ERT), which is further expected to boost the market growth over the forecast period.

Increasing prevalence of pompe disease is expected to boost growth of the market. For instance, according to United Pompe Foundation (UPF), 2017, the estimated frequency occurrence of Pompe disease may vary among different ethnic groups and nationalities: in Holland: 1 in 40,000 (Adults: 1 in 57,000; Infantile: 1 in 138,000), Southern China and Taiwan: 1 in 50,000 births, African-Americans: 1 in 14,000 births and Caucasian: 1 in 100,000 suffer from Pompe disease.

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Gene therapy and enzyme replacement therapy are some of the efficient therapies for the treatment of Pompe disease. Myozyme, a commercialized product in enzyme replacement therapy, is used for the treatment of infants and children with Pompe disease, whereas Lumizyme is used for the treatment of late-onset non-infantile Pompe disease. Key players in the market are focused on developing potential therapies for the treatment of Pompe disease. For instance, NeoGAA GZ402666 by Sanofi and AT-982 by Audentes therapeutics in phase 3 and BMN 701 (Biomarin) in phase 2 clinical trial. This is expected to contribute to the market growth during the forecast period.

However, the treatment of Pompe disease is associated with high cost and long duration, which are expected to hamper the market market growth. For instance, according to a research paper published by Harvard School, in 2016, Lumizyme accounted for US$ 100,000 per year for children and US$ 300,000 per year for adult treatments.

Increasing investments in research and development by manufacturers is expected to propel the market growth

Manufacturers are focusing on expanding their product portfolio. For instance, in September, 2017, Avrobio Inc. expanded their products pipeline with gene therapy for the treatment of pompe disease by utilizing a proprietary lysosomal targeting sequence to deliver high levels of enzyme to lysosomes. Furthermore, in October 2017, Amicus Therapeutics received orphan drug designation to ATB200/AT2221 from the U.S. FDA for the treatment of Pompe disease. This novel treatment paradigm consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P) to enhance uptake, co-administered with AT2221, a pharmacological chaperone.

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Key players operating in the pompe disease therapeutic market include Amicus Therapeutics, Inc., Valerion Therapeutics, LLC, Sanofi S.A., Oxyrane, and Sangamo Therapeutics, Inc.

Table of Content

Global Pompe Disease Therapeutic Market Research Report
Section 1: Global Pompe Disease Therapeutic Industry Overview
Section 2: Global Economic Impact on Pompe Disease Therapeutic Industry
Section 3: Global Market Competition by Industry Producers
Section 4: Global Productions, Revenue (Value), according to Regions
Section 5: Global Supplies (Production), Consumption, Export, Import, geographically
Section 6: Global Productions, Revenue (Value), Price Trend, Product Type
Section 7: Global Market Analysis, on the basis of Application
Section 8: Pompe Disease Therapeutic Market Pricing Analysis
Section 9: Market Chain, Sourcing Strategy, and Downstream Buyers
Section 10: Strategies and key policies by Distributors/Suppliers/Traders
Section 11: Key Marketing Strategy Analysis, by Market Vendors
Section 12: Market Effect Factors Analysis
Section 13: Global Pompe Disease Therapeutic Market Forecast

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