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Pathway Development Consortium announces publication on DMD
The Fly

Pathway Development Consortium announces publication on DMD

The Pathway Development Consortium, a public-private collaboration founded by REGENXBIO Inc. (RGNX) and Solid Biosciences Inc. (SLDB), announced the publication of a peer-reviewed manuscript, Micro-dystrophin expression as a surrogate endpoint for Duchenne muscular dystrophy clinical trials, in Human Gene Therapy. This publication proposes microdystrophin expression levels as a surrogate endpoint reasonably likely to predict clinical benefit. The use of surrogate endpoints reasonably likely to predict clinical benefit could expedite access to therapies for serious diseases that have demonstrated a meaningful advantage over available therapy. Improvements in endpoints that are reasonably likely to provide patients clinical benefit allows patients access while studies are ongoing to verify and describe the predicted clinical benefit to patients under the U.S. Food and Drug Administration accelerated approval pathway. An extended version of the manuscript is available as a white paper on the PDC website and has been submitted to the FDA. This white paper clarifies the rationale for use of the accelerated approval pathway to advance AAV gene therapy development for patients with Duchenne muscular dystrophy and provides support for two surrogate endpoints reasonably likely to predict clinical benefit-muscle fat fraction obtained by magnetic resonance methods and microdystrophin expression levels. "Multistakeholder collaborative efforts that bring together expertise from all backgrounds are critical to bringing new therapeutic options to people with Duchenne," said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy. "The PDC’s white paper on the use of the accelerated approval pathway for AAV gene therapies complements our recent work to update the Community-led Guidance for Dystrophinopathies to advance the development of potential therapies." This white paper expands on the PDC’s draft framework that outlined an approach for the use of FDA’s accelerated approval pathway for different categories of AAV gene therapies that target the underlying monogenic changes that cause disease.

Published first on TheFly

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