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Duchenne muscular dystrophy refers to a severe disorder of a progressive form of muscular dystrophy that primarily affects the males, though in certain rare cases there are incidences in females too. Duchenne muscular dystrophy leads to a progressive weakness and loss, also known as atrophy, in the skeletal and the heart muscles. Some of the early symptoms of the disorder includes a delayed ability to stand, walk or sit and also difficulties in speech. According to the National Organization for Rare Disorders (NORD), it is estimated that Duchenne muscular dystrophy affects 1 in 3,500 male births worldwide.
One of the key drivers of the growth of the duchenne muscular dystrophy treatment was the approval of Eteplirsen in 2016. Also, in June 2019 the Food and Drug Administration (FDA) approved the supplemental New Drug Application (sNDA) for Emflaza (deflazacort) by PTC Therapeutics, Inc., to expand its labelling that will include duchenne muscular dystrophy patients aged between 2 to 5 years. This combined with the strong pipeline of products under clinical trials, are anticipated to drive the growth of the duchenne muscular dystrophy (DMD) treatment market.
Key Market Driver -
Therapeutic approval and strong pipeline of product under development.
Key Market Restraint -
High cost of Duchenne muscular dystrophy treatment.
The factor that is expected to restrain the growth of market is the huge price tag associated with drugs. For example, Eteplirsen (Exondys 51) is expected to cost patients around US$ 300,000 for a treatment course and the cost of the treatment can go as high as US$ 750,000 annually.
Some of the major companies that are present in the global duchenne muscular dystrophy (DMD) treatment market are BioMarin, Bristol-Myers Squibb Company, FibroGen, Inc., Nobelpharma Co., Ltd., NIPPON SHINYAKU CO. LTD., Pfizer Inc., SANTHERA PHARMACEUTICALS, Sarepta Therapeutics and Eli Lilly and Company and other players.
The global duchenne muscular dystrophy (DMD) treatment market has been segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America and Europe are the two largest markets for duchenne muscular dystrophy treatment currently, and are expected to account for a large proportion of the market in the forecast period as well. Rising incidence of duchenne muscular dystrophy, increasing investment in research and development of new treatment options, strong pipeline of products under analysis, are the major factors anticipated to drive the market in these regions.
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In Asia Pacific, Japan is expected to provide the largest market opportunity. In 2017, Japan displayed the drug candidate, DS-5141b, for the Duchenne muscular dystrophy on the country’s SAKIGAKE list, a designation that is equal to the U.S. FDA process of accelerated approval. Many initiatives are being taken by market players to boost the market of Middle East & North Africa. In 2019, CENTOGENE AG announced a year-long strategic collaboration with Sarepta Therapeutics with a goal to increase number of diagnosed patients with duchenne muscular dystrophy in Middle East & North Africa.
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