Duchenne Muscular Dystrophy (DMD): 2018 Market Spotlight Report - 57.5% of Trials in Phase II, and Only 35% and 7.5% of Trials in Phase III and I, Respectively

DUBLIN, May 23, 2018 /PRNewswire/ --

The "Market Spotlight: Duchenne Muscular Dystrophy (DMD)" report has been added to ResearchAndMarkets.com's offering.

This Market Spotlight report covers the Duchenne muscular dystrophy market, comprising key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.

Key Takeaways

• The author estimates that in 2016, there were 179,200 prevalent cases of Duchenne muscular dystrophy in males worldwide, and forecasts that number to increase to 196,200 prevalent cases by 2025.
• Marketed drugs include US FDA-approved drugs such as Emflaza (deflazacort), a glucocorticoid with anti-inflammatory and immunosuppressant properties, and Exondys 51 (eteplirsen), a novel phosphorodiamidate morpholino oligomer designed to induce the skipping of exon 51 in the dystrophin gene. The marketed drugs also include an EU-approved drug, Translarna (ataluren), which acts by targeting premature nonsense mutations.
• Therapies in mid- and late-stage development for Duchenne muscular dystrophy focus on a variety of targets. The majority of pipeline drugs in mid- and late-stage development for Duchenne muscular dystrophy are administered orally, with the remainder including intravenous, intramuscular, intra-arterial, and subcutaneous formulations.
• High-impact upcoming events for drugs in the Duchenne muscular dystrophy space include topline Phase II and Phase III trial results, and a positive CHMP opinion.
• There were 13 licensing and asset acquisition activities involving Duchenne muscular dystrophy drugs during 2013-17, six of which occurred in 2017. The $562m license and collaboration agreement between Sarepta Therapeutics and Summit Therapeutics for European rights to Summit's utrophin modulator pipeline for the treatment of Duchenne muscular dystrophy was the largest deal during the period.
• The clinical trials distribution across Phase I-III indicates that the majority of trials for Duchenne muscular dystrophy have been in Phase II development, with 57.5% of trials in Phase II, and only 35% and 7.5% of trials in Phase III and I, respectively.
• The US has a substantial lead in the number of Duchenne muscular dystrophy clinical trials globally. Italy leads the major EU markets, while Israel has the top spot in Asia.
• Clinical trial activity in the Duchenne muscular dystrophy space is dominated by ongoing trials. PTC Therapeutics has the highest number of ongoing and completed clinical trials for Duchenne muscular dystrophy. - PTC Therapeutics leads industry sponsors with the highest number of clinical trials for Duchenne muscular dystrophy, followed by GlaxoSmithKline.

For more information about this report visit https://www.researchandmarkets.com/research/534m56/duchenne_muscular?w=5

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