- Avidity Biosciences (RNA, Financial) gains FDA accelerated approval pathway for del-brax in FSHD treatment.
- The Phase 3 FORWARD study is initiated to further support global approval.
- FSHD currently affects 45,000-87,000 individuals in the US and Europe, with no approved treatments.
Avidity Biosciences, Inc. (RNA) announced that the U.S. Food and Drug Administration (FDA) has opened the accelerated approval regulatory pathway for del-brax, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD). This pathway allows earlier approval based on surrogate endpoints, potentially expediting the drug's availability to patients. Avidity plans to submit a Biologics License Application (BLA) by the second half of 2026, contingent on data from their ongoing FORTITUDE biomarker cohort.
Simultaneously, Avidity has launched the global Phase 3 FORWARD study, a confirmatory trial designed to assess the long-term effectiveness of del-brax. This 18-month study will involve approximately 200 patients, examining functional mobility, strength, and patient-reported outcomes. The study aims to gather comprehensive data to support del-brax's potential full approval for treating FSHD, a hereditary disorder characterized by progressive muscle weakness.
Del-brax represents a significant advancement in the treatment landscape for FSHD, being the first therapy targeting the DUX4 gene, which causes the disease. FSHD currently affects between 45,000 and 87,000 individuals in the U.S. and Europe, with no FDA-approved treatments available.
CEO of Avidity, Sarah Boyce, stated, "Our commitment to developing del-brax reflects our leadership in rare neuromuscular diseases, moving us closer to providing a transformative treatment option for the FSHD community."
