Pathway Development Consortium Announces Publication in Human Gene Therapy on the Application of FDA’s Accelerated Approval Pathway for AAV Gene Therapies for Patients with Duchenne Muscular Dystrophy
- Publication in Human Gene Therapy provides rationale for the use of microdystrophin expression levels as a surrogate endpoint reasonably likely to predict clinical benefit for AAV gene therapy intended for treatment of patients with Duchenne muscular dystrophy -
- White paper submitted to the
- Expands on Pathway Development Consortium’s previously released white paper that provided a framework for applying the accelerated approval pathway to AAV gene therapy development -
- The work of the
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
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 (FF) obtained by magnetic resonance (MR) 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
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.
“The manuscript and white paper are important steps in providing the scientific rationale that enables use of the accelerated approval pathway to get new treatment options to patients with unmet medical needs,” said
Pathway Development Consortium Contact:
Solid Biosciences Inc.