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(Medical-NewsWire.com, September 17, 2016 ) Highly Innovative and Diverse Pipeline
The Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) pipeline consists of 84 molecules across all stages of development. GBI Research’s analysis revealed a high degree of innovation and diversity in this indication, with 70% of the pipeline being first-in-class products, acting on 13 first-in-class targets. This exceptional first-in-class innovation is largely due to the high number of first-in-class products solely targeting the dystrophin gene, which is the primary genetic cause of DMD and BMD. The strong presence of first-in-class products in the pipeline therefore creates a distinctly different landscape to the market landscape, which relies on symptomatic treatment glucocorticoids. Although Translarna (ataluren) is developed to correct the genetic defects, significant unmet needs remain in the market, as the treatment is applicable to only 10–15% of all DMD cases caused by nonsense mutations.
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Despite a strong focus on personalized treatments that treat the genetic cause of the disease in the DMD/BMD pipeline, innovation is also concentrated on novel molecular targets that alleviate the dystrophic pathology regardless of gene mutations, thereby allowing widespread use in contrast to the mutation-specific treatments. These therapies are expected to be used alongside primary treatment to repair the mutated gene, halt muscle degeneration, and improve life expectancy of patients in the future market.
Strong Alignment of Innovation to Genetics and Disease Processes in Early Pipeline
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Table of Contents
1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3
2 Executive Summary 4
2.1 Highly Innovative and Diverse Pipeline 4
2.2 Alignment of Innovation to Genetics and Disease Processes 4
2.3 Deals Landscape Present Substantial Investment Opportunities 4
3 The Case for Innovation 5
3.1 Growing Opportunities for Biologic Products 6
3.2 Diversification of Molecular Targets 6
3.3 Innovative First-in-Class Product Developments Remain Attractive 6
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 7
3.5 Sustained Innovation 7
3.6 GBI Research Report Guidance 8
4 Clinical and Commercial Landscape 9
4.1 Disease Overview 9
4.2 Disease Epidemiology and Etiology 9
4.2.1 Disease Inheritance 10
4.3 Disease Pathophysiology 10
4.3.1 Hypothesized Pathophysiological Mechanisms of Duchenne Muscular Dystrophy/ Becker Muscular Dystrophy 11
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