Muscular Dystrophy | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2017

Muscular dystrophies (MDs) are a spectrum of genetic disorders characterized by muscle weakness that in severe disease forms can lead to loss of ambulation and early death. MDs share common histological features such as reduction in muscle fiber size, degeneration, and presence of connective and adipose tissue instead of muscle. Duchenne muscular dystrophy (DMD) is the most common subtype with childhood onset, while myotonicdystrophy is the most common in adults. The unmet need for effective treatments that can meaningfully delay or halt progressive muscle degeneration in DMD, as well as in other severe and moderately severe MDs, is high. Ataluren (PTC Therapeutics’ Translarna) targets a genetically defined subset of DMD patients and has been available in some markets following its 2014 European approval; eteplirsen (Sarepta’s Exondys 51) was granted conditional approval in the United States in 2016 and targets a different group of DMD patients who share a similar genetic mutation at the basis of their disease. Though both drugs target the underlying cause of the disease and thus could potentially modify its course, their efficacy as observed in clinical trials thus far appears modest, and the unmet need for effective disease-modifying therapies that would provide relief to all MD patients remains high.

Questions Answered:

  • How large is the diagnosed DMD population in the United States and EU5, and how will its size change over time?
  • What is the current treatment landscape for DMD patients, and how will it change in the next ten years?
  • What is the market share of current agents in the U.S./EU5 DMD market?
  • What do interviewed experts believe is the greatest unmet medical need in the treatment of DMD?
  • What are interviewed experts’ insights on emerging therapies?
  • How will emerging therapies fit into future medical practice?


  • Markets covered: United States, France, Germany, Italy, Spain, and the United Kingdom.
  • Primary research: Six country-specific interviews with thought-leading pediatric neurologists and pediatric cardiologists.
  • Epidemiology: Diagnosed prevalence of DMD patients and drug-treated patients by country segmented by exon skipping pattern and by ambulatory status for the base year 2016 and out years 2021 and 2026. Diagnosed prevalence of Becker muscular dystrophy (BMD) and limb-girdle muscular dystrophy (LGMD).
  • Market forecast: Drug-level sales and patient share of key DMD therapies in 2016 and 2026.
  • Emerging therapies: Phase III/PR: 4 drugs, Phase II: 6; coverage of select Phase I/II and I products.
  • Key companies: AMO Pharma, aTyr Pharma, Akashi Therapeutics, Biogen, Catabasis Pharmaceuticals, EspeRare Foundation, FibroGen, Italfarmaco SpA, Milo Biotechnology, Pfizer, PTC Therapeutics, ReveraGen BioPharma, Roche, Santhera Pharmaceuticals, Sarepta Therapeutics.
  • Key drugs: AAV1-FS344​, ataluren (PTC Therapeutics’ Translarna), BMS-986089, deflazacort (PTC Therapeutics’ Emflaza, generics), domagrozumab, edasalonexent, eteplirsen (Sarepta’s Exondys 51), ezutromid, givinostat, golodirsen, halofuginone delayed release, idebenone, omigapil​, pamrevlumab​, prednisone (generics), rAAVrh74.MCK.micro-Dystrophin, resolaris, rimeporide​, SRP-4045, tideglusib, vamorolone​.
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