Duchenne Muscular Dystrophy | Niche and Rare Pharmacor | G7 | 2015

Publish date: December 2015

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Duchenne muscular dystrophy (DMD) is the most common form of childhood muscular dystrophies. DMD manifests as muscle degeneration and weakness, and it is histologically characterized by features such as reduction in muscle fiber size and degeneration as well as presence of connective tissue and fat instead of muscle. DMD progresses quickly and can lead to loss of ambulation in the early teenage years and subsequent early mortality. Before the conditional approval of ataluren (PTC Therapeutics’ Translarna) in Europe in August 2014, glucocorticoid treatment was the only available treatment for DMD patients. The launch of ataluren for the treatment of DMD stemming from nonsense mutations in the dystrophin gene signals a new era of disease-modifying therapies (DMTs) for DMD, as eteplirsen (Sarepta Therapeutics) and agents earlier in development from Sarepta, which also target the underlying cause of DMD, may become available in the near future.

Questions Answered in This Report:

  • DMD is a rare genetic disease affecting only a small percentage of the population. What is the size of the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom) diagnosed prevalent population for DMD? How will population size change through 2024?

  • Glucocorticoid therapy, mainly with prednisone, prednisolone, or deflazacort, is the cornerstone of current therapy options for DMD owing to clinical trials demonstrating that this class of agents decreases the progressive loss of muscle strength and function in DMD patients. What other treatments are used in DMD, and how will treatment of DMD change through over the next ten years?

  • Unmet needs in DMD are many and span a range of challenging issues. What are the most promising avenues of R&D? Which unmet needs do thought leaders expect to remain unaddressed by mid- to late-stage emerging therapies?

  • Determining appropriate primary end points is a challenge in DMD trials. What is expert opinion of the six-minute walk test, a primary end point commonly used in DMD trials of ambulatory patients?

  • We forecast that four new therapies for DMD will reach the U.S. and major European markets by 2024. Which treatments will target the underlying cause of the disease? What percentage of the DMD population will be eligible for these treatment options? What will be the therapeutic landscape in 2024?

Scope:

Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: 8 country-specific interviews with DMD experts.

Epidemiology: Diagnosed prevalence of DMD.

Emerging therapies: Phase III: 4; Phase II: 11; coverage of select preclinical and Phase I products.

Author(s): Matthew Mackenzie, M.A.
Kristin Dorfman, M.P.H., M.S.