Our Progress
Accelerated Approval for Duchenne AAV Gene Therapies
Applying the Accelerated Approval Pathway to AAV Gene Therapy for Treatment of Duchenne Muscular Dystrophy discusses the rationale for use of the accelerated approval pathway to advance AAV gene therapy development for patients with Duchenne and identifies two surrogate endpoints reasonably likely to predict clinical benefit—muscle fat fraction (FF) obtained by magnetic resonance (MR) methods and microdystrophin. It also discusses aspects that are important for approval of medical products generally and others that are unique to medical products pursuing accelerated approval.
Accelerating AAV Gene Therapy Development
Draft Framework for AAV Gene Therapy Development proposes a framework that can be applied to AAV gene therapies that would facilitate the use of the accelerated approval pathway at the US Food and Drug Administration. It identifies different categories of AAV gene therapies that target the underlying monogenetic changes that cause disease, and proposes generalized approaches that clarify the evidence needed to support an approval.
Feedback to the proposed framework, drafted by REGENXBIO and Solid Biosciences, can be sent to info@pathwaydevelopmentconsortium.org.
Duchenne Priorities
Advancing AAV Gene Therapy for Duchenne Muscular Dystrophy builds on the discussions at the PDC’s initial roundtable on Duchenne and identifies areas where attention is needed to facilitate development of AAV gene therapies for Duchenne. The PDC seeks to work collaboratively to address these issues in the hopes to bring more therapeutic options to patients.
Duchenne Roundtable Summary
AAV Gene Therapy for Duchenne Muscular Dystrophy: Finding a path forward for meaningful clinical endpoints in clinical trials summarizes the discussions at the Duchenne Roundtable hosted by the PDC on April 6, 2021. The discussions focused on the challenges and opportunities in AAV gene therapy development for Duchenne, including patient/caregiver preferences, clinical trial design, and appropriate endpoints to assess clinical benefit.