Thursday 30 April 2015, Amsterdam
The report 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
The report 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.
The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.