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Market Spotlight: Duchenne Muscular Dystrophy (DMD), Pharma Intelligence

Market Spotlight: Duchenne Muscular Dystrophy (DMD), Pharma Intelligence

  • November 2019
  • 61 pages
  • ID: 5780150
  • Format: PDF
  • By Pharma Intelligence


Table of Contents

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

Key Takeaways
The analyst estimates that in 2017, there were 182,100 prevalent cases of Duchenne muscular dystrophy (DMD) in males worldwide, and forecasts that number to increase to 199,100 prevalent cases by 2026.
Marketed drugs for DMD include the FDA-approved drugs Emflaza, a glucocorticoid with anti-inflammatory and immunosuppressant properties, and Exondys 51, 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, which acts by targeting premature nonsense mutations. Emflaza and Translarna are administered via the oral route, while Exondys 51 is available as an intravenous formulation.
The largest proportion of industry-sponsored drugs in active clinical development for DMD are in Phase II, with two drugs in the NDA/BLA phase.
Therapies in development for DMD focus on a wide variety of targets. The majority of pipeline drugs are administered via the intravenous or oral routes, with the remainder being intramuscular, intraarterial, and subcutaneous formulations.
High-impact upcoming events for drugs in the DMD space comprise topline Phase II and Phase III trial results, and an expected PDUFA date for an NDA.
The overall likelihood of approval of a Phase I single-gene disorders (non-inborn errors of metabolism) asset is 23.9%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 7.2 years from Phase I to approval, compared to 8.8 years in the overall metabolic space.
There have been 21 licensing and asset acquisition deals involving DMD drugs during 2014–19, eight of which occurred in 2017. The largest deal during the period was the $3,520m licensing agreement between Vertex and CRISPR Therapeutics signed in 2017, as part of which the two companies will discover and develop gene editing therapies for the treatment of DMD and myotonic dystrophy type 1 (DM1).
The distribution of clinical trials across Phase I–IV indicates that the majority of trials for DMD have been in the early and midphases of development, with 70% of trials in Phase I–II, and only 30% in Phase III–IV.
The US has a substantial lead in the number of DMD clinical trials globally. The UK leads the major EU markets, while Israel has the top spot in Asia.
Sarepta Therapeutics has the highest number of ongoing trials for DMD, with nine trials.
PTC Therapeutics leads industry sponsors with the highest number of clinical trials for DMD, followed by Sarepta Therapeutics.

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