Solid Biosciences Wins EU Orphan Drug Status for Friedreich's Ataxia Gene Therapy SGT-212

Solid Biosciences Inc. (SLDB) said the European Commission granted Orphan Medicinal Product designation to SGT-212, its investigational gene therapy for Friedreich's ataxia, a rare inherited disease causing progressive neurological damage and cardiac dysfunction. The company reported five participants dosed in the Phase 1b FALCON trial as of October 5, 2026, with no treatment-related serious adverse events observed at that time. SGT-212 delivers the full-length frataxin gene via MRI-guided delivery to the cerebellar dentate nuclei combined with intravenous administration targeting the heart and other affected tissues. The open-label trial evaluates safety and tolerability in adults aged 18 to 40. SGT-212 also holds Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. Solid Biosciences expects initial FALCON data in the first quarter of 2027. SLDB traded between $3.79 and $11.45 over the past year and was recently at $5.83, down 1.52%.