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

In a press release on January 12, 2026, Solid Biosciences announced that U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to SGT-212 for the treatment of Friedreich’s ataxia (FA). The FDA grants Orphan Drug designation to investigational therapies intended for the treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.

Learn more about the SGT-212 program on our pipeline page.