Muscular Dystrophy | Niche and Rare Pharmacor | G7 | 2014

Muscular dystrophy (MD) is associated with degeneration and weakness of affected muscles that vary with the type of dystrophy. Dystrophies are characterized by common histological features such as reduction in muscle fiber size and degeneration as well as presence of connective tissue and fat instead of muscle. Traditionally, dystrophies are classified according to age at onset, inheritance, and affected muscles. Several types of clinically and genetically distinct dystrophies have been identified. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy occurring in children; it progresses quickly and can lead to loss of ambulation in the early teenage age years and subsequent early mortality. Prior to the conditional approval of ataluren (PTC Therapeutics’ Translarna) in the European Union in August 2014, glucocorticoid treatment was the only available treatment for DMD patients. The launch of Translarna for the treatment of DMD stemming from nonsense mutations in the dystrophin gene will usher in a new era of disease-modifying therapies (DMTs) for MD.

This report provides an overview of the MD market including a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. The report identifies a very high level of unmet need for new DMTs for MDs. Interviewed experts eagerly await the launch of emerging exon-skipping agents from Prosensa and Sarepta and are hopeful that these new therapeutic approaches will allow them to fundamentally alter the disease course for many of their patients. The findings described in this report derive from detailed interviews with expert U.S. and European specialists, secondary research, and best-in-class epidemiological analysis. This report provides deep insights into this complex and evolving clinical space and includes a detailed analysis of specific opportunities for current and emerging therapies, including experts’ views on which unmet needs have not yet been addressed by developers of new therapies.