Friedreich's Ataxia Treatment Progresses as Gene Therapy Candidate Earns Regulatory Recognition

Solid Biosciences Inc. (SLDB), a developer specializing in therapies for neuromuscular and cardiac disorders, has revealed that its investigational gene therapy SGT-212 has received Orphan Drug designation from the U.S. Food and Drug Administration. The milestone recognition underscores the therapeutic potential of SGT-212 in addressing Friedreich’s ataxia, a rare genetic neurological condition.

Clinical Trial Advancement and Treatment Mechanism

The company has completed enrollment of its first participant in the FALCON Phase 1b study, designed to evaluate the safety profile and tolerability of SGT-212 in patients aged 18 to 40 who have been diagnosed with Friedreich’s ataxia accompanied by cardiac hypertrophy. Preliminary findings from this open-label, multi-center trial are anticipated to emerge during the latter half of 2026, pending continued participant recruitment.

SGT-212 represents a recombinant AAV-based gene replacement approach utilizing an innovative dual-route delivery system. The therapeutic strategy combines direct infusion into the intradentate nuclei—a targeted brain region within the cerebellum—with subsequent intravenous administration into systemic circulation. This coordinated delivery mechanism enables the therapy to address neurologic, cardiac, and systemic manifestations of the disease that contribute to patient morbidity and mortality.

Regulatory Pathway and Disease Context

Prior to receiving the Orphan Drug designation, SGT-212 had already achieved two significant regulatory milestones: Fast Track Designation status granted on January 21, 2025, and Rare Paediatric Disease Designation conferred on December 1, 2025. These designations reflect regulatory recognition of the unmet medical need in this patient population.

Friedreich’s ataxia, affecting approximately 5,000 individuals in the United States and around 15,000 across Europe, stems from a genetic mutation within the FXN gene. This mutation results in diminished frataxin protein levels, a cofactor critical for proper mitochondrial function. Currently, no curative treatment exists for the condition; therapeutic options are limited to disease-modifying interventions.

Broader Pipeline and Market Performance

Solid Biosciences maintains a diversified portfolio of rare disease gene therapy candidates. The pipeline includes SGT-003 targeting Duchenne muscular dystrophy, SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy, and additional programs addressing fatal genetic neuromuscular and cardiac conditions.

In market activity, SLDB shares closed at $5.24, reflecting a decline of 0.95% during the trading session.