Trial Profile

A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping

Status: Active, no longer recruiting

Phase of Trial: Phase II

Latest Information Update: 09 Aug 2026

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At a glance

30 Jul 2026 Planned End Date changed from 31 Jul 2027 to 30 Apr 2027.
21 Jul 2026 According to a Novartis media release, A Biologics License Application (BLA) was submitted to the FDA for accelerated approval of del-zota in people living with Duchenne muscular dystrophy (DMD) who have a genetic variant that may be amenable to exon 44 skipping (DMD44).
19 Nov 2025 According to an Avidity Biosciences media release, company aligned with FDA on a path forward for a BLA submission for del-zota following an October 2025 pre-BLA meeting, with the submission planned for 2026 for accelerated approval.

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