Global Pompe Disease Treatment Market – Industry Trends and Forecast to 2025

Market Overview

Pompe disease is a genetic metabolic disorder that occurs in infants and is caused due to the mutations in the GAA gene responsible for producing the acid, alpha-glucosidase enzyme that converts glycogen into a simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and results to have a heart problem, muscle weakness, liver damage that can lead to premature death in the new-born.

Global Pompe Disease Treatment Marketis expected to register aCAGR of3.0%during the forecast period of2019 to 2030and was valued atUSD1,181.6 Millionin 2018.

The demand for Pompe disease treatment is increasing due to the increasing government initiatives and rising R&D activities by manufacturers. However, the high cost of treatment is hampering the market growth.

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The rising prevalence of Pompe disease is creating opportunities for manufacturers to find new solutions for the diagnosis and treatment. According to the National Organization for Rare Disorders, the estimated frequency occurrence of Pompe disease is approximately 1 in 40,000 people in the US. Currently, manufacturers are focusing on expanding their pipeline to cater to the increasing demand for Pompe disease treatment. For instance, in September 2017, AVROBIO’s initiated a pre-clinical project which includes third gene therapy to treat lysosomal storage disorders by utilizing lysosomal targeting sequence to deliver high levels of enzymes to lysosomes. This project helped the company to expand its research & development pipeline and develop effective products for the treatment of Pompe disease.

Governments in various countries are taking continuous initiatives for developing and manufacturing innovative products for the diagnosis & treatment of rare diseases such as Pompe disease. For instance, in the US the Orphan Product Grants funded 18 new grants from a total of 92 grant applications in 2015 to support around 67 ongoing clinical studies that are related to rare diseases.

Segmentation

The hospitals & clinics segment was valued at USD 860.8 million in the year 2018, and the research & academic institutes segment accounted for a value of USD 320.7 million in 2018 and are expected to exhibit a CAGR of 2.3% during the forecast period.

Global Pompe Disease Treatment Market, by Type

• Late-Onset Pompe Disease
• Classic Infantile-Onset Pompe Disease
• Non-Classic infantile-onset Pompe Disease

Global Pompe Disease Treatment Market, byTherapy

• Enzyme Replacement Therapy
• Gene Therapy
• Others

Global Pompe Disease Treatment Market, byEnd User

• Hospitals and Clinics
• Research and Academic Institutes

Regional Analysis

The Global Pompe Disease Treatment Market, based on region, has been divided into the Americas, Europe, Asia-Pacific, and the Rest of the World.

The Americas are likely to dominate the global Pompe disease treatment market owing to the presence of established payers and an increase in the number of Pompe disease patients in the region. In addition, the presence of a huge number of hospitals in the region also would support the growth of the market during the forecast period. According to a study published by the American Hospital Association in February 2018, there were 5,534 registered hospitals in the US in 2017.

Europe held a substantial share in the global Pompe disease treatment market. This can be attributed to increasing research & development initiatives; and government support for improving the healthcare sector.For instance, European countries are extremely cautious and have implemented the newborn screening (NBS) program as a means of early detection and identification of severe illnesses in newborns.

Asia-Pacific is estimated to be the fastest-growing region in the global market. This is due to the increasing healthcare expenditure, growing awareness among patients regarding rare disorders, and expanding the healthcare industry in this region.

The Pompe disease treatment market in the Rest of the World is expected to witness steady growth during the review period owing to growing awareness about the treatment of rare diseases and developing healthcare infrastructure.

Key Players

The Prominent Players in the Global Pompe Disease Treatment Market areAmicus Therapeutics, Inc (US) Sanofi (France), Audentes Therapeutics (US), Oxyrane (UK), Valerion Therapeutics (US), AVROBIO, Inc. (US), CENTOGENE AG (UK).

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