Increasing government initiativesis expected to augment growth of the pompe disease therapeutic market
Furthermore, initiatives from thegovernments of economies such as Sweden, the Netherlands, and South Korea thatfocus on approving guidelines for the reimbursement of individuals undergoingenzyme replacement therapy (ERT) are also expected to boost growth of the PompeDisease Therapeutic Market in near future. Considering the public healthproblems, government is taking continuous initiatives in promoting andadvancing the development of innovative products for the prevention anddiagnosis or treatment of rare diseases or conditions such as Pompe disease.For instance, in 2015, Orphan Product Grants Program funded 18 new grant awardsfrom 92 grant applications and provided funding and support to around 67ongoing clinical study projects related to rare disease.
Pompe disease is an inheritedLysosomal Storage Disorder (LSD) caused by a deficiency of enzyme acid namedalpha-glucosidase (GAA). This causes the glycogen to accumulate in differentkinds of tissues, primarily into smooth muscles, skeletal muscles and cardiacmuscle. It leads to muscle weakness, respiratory problems, and may also affectsthe liver and heart. Pompe disease is inherited as an autosomal recessivegenetic trait. There are three major types of pompe disease, which differ inseverity and appear at different age, these types are known as classicinfantile-onset, which starts within a few months of birth, non-classicinfantile-onset Pompe disease- usually appears by age 1, and late-onset type ofPompe disease that may shows the symptoms later in childhood, adolescence, oradulthood. Pompe disease therapeutic market can exhibit immense growthpotential due to presence of limited number of manufacturers, which cansignificantly enhance and expand their business potential and scope in thismarket.
* The sample copy includes: Report Summary, Table ofContents, Segmentation, Competitive Landscape, Report Structure, Methodology.
Request a sample copyof this report: https://www.coherentmarketinsights.com/insight/request-sample/1316
Increasing prevalence of pompedisease in many economies is expected to have a lucrative opportunities forpharmaceutical manufacturers to come with a tailored solution for diagnosis andtreatment of disease. For instance, according to United Pompe Foundation (UPF),2017, the estimated frequency occurrence of Pompe disease may vary amongdifferent ethnic groups and nationalities: in Holland: 1 in 40,000 (Adults: 1in 57,000; Infantile: 1 in 138,000), Southern China and Taiwan: 1 in 50,000births, African-Americans: 1 in 14,000 births and Caucasian: 1 in 100,000suffer from pompe disease. Gene therapy and enzyme replacement therapy may behighly effective at reducing clinical manifestations of this rare disease.Currently, there are two commercialized products in enzyme replacement therapy,which are Myozyme for the treatment of infants and children with Pompe diseaseand Lumizyme for late-onset non-infantile Pompe disease. Furthermore, variouspipeline products in phase 3 (NeoGAA GZ402666 (Sanofi), AT-982 (Audentestherapeutics)) and in phase 2 (BMN 701(Biomarin)) clinical trial are alsoexpected to create a meteoric growth in this market during the forecast period.However, the high cost associated with the treatment and long duration of thetreatment are the factors restraining growth of the pompe disease therapeuticmarket. For instance, according to a research paper published by HarvardSchool, in 2016, Lumizyme accounted for US$ 100,000 per year for children andUS$ 300,000 per year for adult treatments.
Browse ResearchReport: https://www.coherentmarketinsights.com/ongoing-insight/pompe-disease-therapeutic-market-1316
Increasing investments inresearch and development by manufacturers is expected to propel the marketgrowth
Manufacturers are focusing onfinding new solutions by expanding their pipeline to cater the demand for pompedisease therapeutics market. For instance, in September, 2017, Avrobio Inc.expanded their products pipeline with gene therapy to treat pompe disease byutilizing a proprietary lysosomal targeting sequence to deliver high levels ofenzyme to lysosomes. Furthermore, in October 2017, Amicus Therapeutics receivedorphan drug designation to ATB200/AT2221 from the U.S. FDA for the treatment ofPompe disease. This novel treatment paradigm consists of ATB200, a uniquerecombinant human acid alpha-glucosidase (rhGAA) enzyme with optimizedcarbohydrate structures, particularly mannose-6 phosphate (M6P) to enhanceuptake, co-administered with AT2221, a pharmacological chaperone.
Key players operating in thepompe disease therapeutic market include Amicus Therapeutics, Inc., ValerionTherapeutics, LLC, Sanofi S.A., Oxyrane, and Sangamo Therapeutics, Inc.
Buy-Now this researchreport: https://www.coherentmarketinsights.com/insight/buy-now/1316
AboutCoherent Market Insights:
CoherentMarket Insights is a prominent market research and consulting firm offeringaction-ready syndicated research reports, custom market analysis, consultingservices, and competitive analysis through various recommendations related toemerging market trends, technologies, and potential absolute dollaropportunity.
ContactUs:
mailto:sales@coherentmarketinsights.com
U.S.Office:
Name: Mr. Shah
CoherentMarket Insights 1001 4th Ave,
# 3200Seattle, WA 98154, U.S.
US : +1-206-701-6702
UK : +44-020-8133-4027
JAPAN: +050-5539-1737
