This market research report presents a detailed analysis of the key drivers, challenges, and upcoming trends that influence the prospects for growth in this market. Leading vendors analyzed in this market research are Amicus Therapeutics, BioMarin Pharmaceutical, and Genzyme.
Technavio has released its new market research report, Pompe disease market in the US, which is expected to grow at a CAGR of around 11% between 2015 and 2019. This market is expected to grow rapidly during the forecast period owing to the special provisions allocated for orphan drugs. These drugs are drugs are provided with tax credits for qualified clinical testing, exemption from prescription drug user fees and market exclusivity for seven years.
“Governments and NGOs across the world are organizing various events and fund-raising programs to create awareness about this disease. Organizations such as the United Pompe Foundation, International Pompe Association, Canadian Association of Pompe, Acid Maltase Deficiency Association, and NORD are educating people online about the various treatment oprions available for this disease. Also, these organizations are providing financial support to the patients through favourable reimbursement policies,” says Imran Mushtaq, Lead Analyst, Healthcare & Lifesciences, Technavio Research.
The diagnosis of the Pompe disease is quite challenging as its signs and symptoms are similar to other muscular, gastrointestinal, or respiratory disorders. The infantile-onset Pompe disease generally manifests itself within a few months after the baby is born and is characterized by symptoms like myopathy, hypotonia, hepatomegaly, and a defective heart. The adult-onset Pompe disease generally manifests itself during adolescence or adulthood. This disease is generally milder than its infantile-onset form and is characterized by symptoms like progressive muscle weakness in the legs and the trunk.
The leading vendors in the Pompe disease market in the US include Amicus Therapeutics, BioMarin Pharmaceutical, and Genzyme. This market is highly lucrative and is characterized by the presence of a limited number of vendors. Out of all the vendors in this market, only Genzyme has produced an approved drug for the treatment of the disease. Vendors that have the capability to manufacture and market a drug that can effectively treat Pompe disease and also exhibit a high efficacy profile are expected to gain maximum traction in the market.
A more detailed analysis is available in the Technavio report, Pompe Disease Market in the US 2015-2019.
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