PRO38 Twenty Years of Clinical Trials in Duchenne Muscular Dystrophy: A Low Clinical Drug Development Success

Dovari, A.; Inuganti, B.; Nadimpally, J; Vatturi, S.M.; Hyderboini, RK; Goyal, Richa

doi:10.1016/j.jval.2021.04.1023

Cited by 2 publications

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“…Despite almost three decades of preclinical and clinical research, no treatment other than steroids has formally demonstrated clinical efficacy in DMD. The attrition rate between early-phase and late-phase development is high ( Dovari et al, 2021 ). Several developments have been discontinued due to inefficacy despite early-phase positive results or signals of efficacy in secondary outcomes.…”

Section: Discussionmentioning

confidence: 99%

Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy

et al. 2021

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Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of functional dystrophin protein. Patients experience progressive muscle weakness, cardiomyopathy and have a decreased life expectancy. Standards of care, including treatment with steroids, and multidisciplinary approaches have extended the life expectancy and improved the quality of life of patients. In the last 30 years, several compounds have been assessed in preclinical and clinical studies for their ability to restore functional dystrophin levels or to modify pathways involved in DMD pathophysiology. However, there is still an unmet need with regards to a disease-modifying treatment for DMD and the attrition rate between early-phase and late-phase clinical development remains high. Currently, there are 40 compounds in clinical development for DMD, including gene therapy and antisense oligonucleotides for exon skipping. Only five of them have received conditional approval in one jurisdiction subject to further proof of efficacy. In this review, we present data of another 16 compounds that failed to complete clinical development, despite positive results in early phases of development in some cases. We examine the reasons for the high attrition rate and we suggest solutions to avoid similar mistakes in the future.

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Section: Discussionmentioning

confidence: 99%

Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy

et al. 2021

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“…Despite almost three decades of preclinical and clinical research, no treatment other than steroids has formally demonstrated clinical efficacy in DMD. The attrition rate between earlyphase and late-phase development is high (Dovari et al, 2021). Several developments have been discontinued due to inefficacy despite early-phase positive results or signals of efficacy in secondary outcomes.…”

Section: Discussionmentioning

confidence: 99%

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PRO38 Twenty Years of Clinical Trials in Duchenne Muscular Dystrophy: A Low Clinical Drug Development Success

Cited by 2 publications

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Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy

Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy

Presentation1.pdf

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