FDA opens accelerated approval pathway for Avidity's del-brax in FSHD; positive Phase 1/2 data

Avidity Biosciences, Inc.

FDA confirmed accelerated approval pathway for del-brax; BLA submission targeted H2 2026.
Phase 3 FORWARD study initiated: 200 patients, 45 sites globally, 18-month placebo-controlled.
Phase 1/2 FORTITUDE dose-escalation cohorts (39 patients) showed improvement in mobility, strength, and quality of life vs placebo.
Rapid reductions in KHDC1L and creatine kinase; no related serious AEs or discontinuations.
Biomarker cohort topline data expected Q2 2026; primary endpoint KHDC1L reduction.

item 7.01item 8.01item 9.01

This headline and bullets were generated automatically by deepseek-v4-flash:cloud@v2 from the public filing. Read the source on SEC.gov before relying on any specific claim. Not investment advice. See methodology for how this pipeline works.