Muscular Dystrophy | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2019

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. Duchenne muscular dystrophy (DMD) is the most common subtype with childhood-onset, while myotonic dystrophy is the most common subtype with adult-onset. 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), which targets DMD patients with nonsense dystrophin mutations, has been available in some European markets for DMD following its conditional approval by the EMA in 2014; and eteplirsen (Sarepta’s Exondys 51), which targets DMD patients with dystrophin mutations amenable for skipping of exon-51, has been available in the United States since 2016, when it was granted conditional approval for DMD. Though both drugs target the underlying cause of disease in DMD and, thus, could modify its course, their efficacy as observed in clinical trials appears modest. We anticipate the launch of two new potentially disease-modifying therapies—Sarepta Therapeutics’ exon-skipping agents golodirsen and casimersen—and two nonspecific symptomatic therapies—Santhera Pharmaceuticals’ idebenone and Italfarmaco SpA’s givinostat—during the 2017-2027 forecast period. Despite the launch of four new agents, the unmet need for effective pharmacotherapies for DMD will remain high.

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