Global Pompe Disease Market 2015-2019
About Pompe Disease
Pompe disease is a type of LSD. LSD refers to a group of diseases arising from the accumulation of specific substrates. LSDs are progressive and have variable age of onset and clinical symptoms. LSDs are inherited autosomal recessive disorders. Pompe disease has two broad sub-types: infantile-onset Pompe disease and adult-onset Pompe disease. Pompe disease occurs as a result of mutations in the GAA gene. These mutations are responsible for the preventing the action of the enzyme acid alpha-glucosidase, which is responsible for catabolizing glycogen, thereby allowing sugar to accumulate and reach toxic levels in the lysosomes of cells. The buildup of glycogen in certain organs and tissues, mostly in the muscles, hinders the normal functions of organs. Pompe disease is an autosomal recessive disease. This implies that both the parents pass on one abnormally mutated copy of the gene to their child. A parent carrying an abnormal copy of the gene is known as a carrier. Pompe disease affects both men and women, irrespective of the gender. Pompe disease affects people across all ethnicities and races. Individuals affected with Pompe disease acquire the disease at birth, however, the onset of symptoms varies extensively. More than 300 genetic mutations that can cause Pompe disease have been identified by the researchers till date. Though there is no cure for Pompe disease, diet therapy and enzyme replacement therapy are effective in reducing the clinical manifestations of the disease.
TechNavio's analysts forecast the Global Pompe Disease market to grow at a CAGR of 11.79 percent over the period 2014-2019.
Covered in this Report
This report covers the present scenario and the growth prospects of the Global Pompe Disease market for the period 2015-2019. To calculate the market size, the report considers revenue from the sales of drugs available in the market for the management of Pompe disease. The report also presents the vendor landscape and a corresponding detailed analysis of the major vendors in the Global Pompe Disease market. The report also considers the revenue that is likely to be generated from the drug candidates that are under various phases of development. In addition, it discusses the major drivers that influence the growth of the market, the challenges faced by the vendors and the market at large, as well as the key trends that contribute to the growth the market. TechNavio's report, Global Pompe Disease Market 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the Americas, and the EMEA and APAC regions; it also covers the Global Pompe Disease market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.Key Regions
Technavio Announces the Publication of its Research Report – Global Pompe Disease Market 2015-2019
Technavio recognizes the following companies as the key players in the Global Pompe Disease Market: Amicus Therapeutics Inc., BioMarin Pharmaceutical Inc. and Genzyme Corp
Other Prominent Vendors in the market are: Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences and Valerion Therapeutics
Commenting on the report, an analyst from Technavio’s team said: “Increased awareness has made regulatory authorities work steadily toward developing guidelines and providing assistance to drug manufacturers to promote R&D of drugs to treat the disease. Emerging economies are rapidly becoming aware of the importance of orphan drug designations and are working toward the development of regulatory protocols required to award these designations.”
According to the report, special provisions allocated to drugs for orphan diseases have a positive impact on market growth. Incentives in the form of funding, guidance, and marketing exclusivity provide attractive opportunities to vendors to conduct research in this disease area. For instance, the drug Myozyme was designated as an orphan drug by the FDA in 1997.
Further, the report states that a limited patient population hinders the effective evaluation of drugs based on clinical trials. The presence of few affected people has a negative impact on drug sales and prolongs the time period by which a vendor can recover the R&D expenses of the drug.
Amicus Therapeutics, BioMarin Pharmaceutical, Genzyme , Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences , Valerion Therapeutics