Global Duchenne Muscular Dystrophy Treatment Market
Duchenne muscular dystrophy (DMD) is a genetic disorder and it is one of the 9 types of muscular dystrophy, which is characterized by progressive degeneration and weakness of muscle. It is caused due to the absence of dystrophin protein which helps to keep muscle cells intact. Onset of symptoms will be from early childhood in the age of 3-5. Guillaume Benjamin Amand Duchenne was the first French neurologist to describe about Duchenne muscular dystrophy in 1860’s.There is no known cure for duchenne muscular dystrophy but the treatment is given to control the symptoms to maximize the quality of life of the patient.
Increase in the prevalence rate of duchenne muscular dystrophy is the major factor expected to drive the market growth of Market. As per, NHS estimation 100 are born with duchenne muscular dystrophy every year in the UK, while 349 new cases were reported in the U.S. in 2010 as per to Centre for Disease Control. Furthermore, Increasing in healthcare expenditure and growing awareness about Duchenne muscular dystrophy is also propelling the growth of the Market. However, low treatment seeking rate because of delayed diagnosis and fast prognosis of the disorder are the factors which are restraining the growth of Duchenne muscular dystrophy.
The Duchenne Muscular Dystrophy Treatment Market is classified on the basis of drug class, infection type distribution channel, and geography.
Based on drug class, the Global Market is segmented into the following
- Pain management
Based on distribution channel, the Global Market is segmented into the following
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
There are very few products are marketed for the treatment of duchenne muscular dystrophy-like Translama, Exodus 51 and Emflaza. Presently this market is in research phase majorly and focuses on the on-going clinical trials for the development of innovative products. Government organizations are focussing on the prevention of duchenne muscular dystrophy. For instance, world Duchenne awareness day was initiated by The United Parent Projects Muscular Dystrophy (UPPMD) with the motto of rising awareness regarding duchenne muscular dystrophy globally and they have declared 07 th September as the world’s duchenne awareness day. In 2015, U.S. FDA has published a specific guidance for the development of drugs to treat duchenne muscular dystrophy. Innovative approaches like utrophin modulation which is going to launch during the forecast period to treat DMD and approvals for the drugs such as ezutromid of summit therapeutics a phase II clinical trial drug which received orphan drug designation fast approval from U.S.FDA and BioMarin Pharmaceuticals is developing an exon-skipping drisapersen which is phase III clinical trial evaluation all these developments are expected to offer lucrative opportunities for Duchenne Muscular Dystrophy Treatment Market.
On the basis of geographical regions, Duchenne Muscular Dystrophy Treatment Market is classified as into five key regions as Europe, Asia- Pacific, North America, Middle East & Africa and Latin America. The European region is the dominating market in global DMD treatment market owing to the several treatments for DMD which are approved and are under review in EU. For instance, PTC Therapeutics has recently received orphan drug designation and conditional approval for its Translarna, a gene therapy to treat DPD. North America holds a significant position in DMD treatment market owing to the high prevalence of DMD. Emerging markets like Asia Pacific, Latin America are expected to have significant growth rate owing to the growing prevalence of DMD, large patient pool and growing healthcare expenditure and awareness of DMD.
Some of the players in Duchenne Muscular Dystrophy Treatment Market are Bristol-Myers Squibb(U.S), FibroGen, Inc (U.S), ITALFARMACO S.p.A. (Italy), Catabasis Pharmaceuticals, Inc.(U.S) , NS Pharma, Inc. (U.S), Marathon Pharmaceuticals, LLC (U.S), ReveraGen BioPharma, Inc. (U.S), Pfizer (U.S), Summit Therapeutics plc (U.K), Taiho Pharmaceutical Co (Japan), Sarepta Therapeutics (U.S), Inc., Santhera Pharmaceuticals, PTC Therapeutics (Switzerland).
- In 2017 March, PTC Therapeutics entered into the purchase agreement with Marathon Pharmaceuticals for its Emflaza to treat DMD.
- In February 2017, deflazacort (Emflaza) of marathon pharmaceuticals (Acquired by PTC Therapeutics) to treat DMD has received U.S.FDA approval.
- In September 2016, U.S.FDA granted accelerated approval to Sarepta Therapeutics Eteplirsen (Exondys 51) a disease-modifying drug for treating DMD
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