Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich's Ataxia

Core Insights - Solid Biosciences has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations for SGT-212, a gene therapy for Friedreich's ataxia (FA) [1][2] - The first participant has been dosed in the Phase 1b FALCON trial, with initial data expected in the second half of 2026 [1] Regulatory Designations - The Orphan Drug designation highlights the significant unmet need in the FA community and recognizes the therapeutic potential of SGT-212's dual-route administration [2] - These designations may help streamline and accelerate the development path for SGT-212 [2][3] FALCON Trial Details - FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial evaluating the safety and tolerability of SGT-212 in participants aged 18-40 diagnosed with FA and cardiac hypertrophy [4] - The trial is being conducted in the United States [4] SGT-212 Overview - SGT-212 is a recombinant AAV-based gene replacement therapy designed to deliver full-length human frataxin via a dual route: intradentate nucleus infusion followed by intravenous infusion [5] - The therapy aims to restore therapeutic levels of frataxin to address the neurological, cardiac, and systemic manifestations of FA [5] Friedreich's Ataxia (FA) Background - FA is a life-threatening, degenerative multisystem disease affecting approximately 5,000 people in the U.S. and 15,000 in Europe, with no current treatments available to cure or halt disease progression [6]