Solid Biosciences announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its SGT‑212 dual‑route gene‑therapy program for Friedreich’s ataxia, and that the first participant in the Phase 1b FALCON trial has been dosed. The designation, which also includes Fast Track and Rare Pediatric Disease status, is intended to streamline development and potentially accelerate approval for a treatment of a disease that affects fewer than 200,000 people in the United States.
The Orphan Drug Designation was awarded on January 12 2026, while Fast Track status was granted on January 21 2025 and Rare Pediatric Disease status on December 1 2025. These designations provide seven years of market exclusivity upon approval, tax credits, fee waivers, and the possibility of a pediatric priority review voucher, all of which can reduce development costs and shorten the time to market.
The FALCON trial is a Phase 1b study that evaluates the safety and tolerability of SGT‑212, which delivers full‑length frataxin via a stereotactic, MRI‑guided intradentate nuclei infusion followed by an intravenous infusion. Early data are expected in the second half of 2026, contingent on enrollment. The dual‑route delivery is designed to address both central nervous system and cardiac manifestations of Friedreich’s ataxia, a key differentiator from other gene‑therapy candidates that target only one organ system.
Friedreich’s ataxia affects approximately 5,000 people in the United States and 15,000 in Europe, with a prevalence of about 1 in 50,000 in the U.S. The disease’s rarity qualifies it for orphan status, and the small patient population underscores the importance of accelerated development pathways and the potential for significant unmet medical need. The designation also signals that Solid Biosciences is expanding its pipeline beyond its Duchenne muscular dystrophy program into a broader rare‑disease portfolio.
The gene‑therapy landscape for Friedreich’s ataxia is competitive, with Lexeo Therapeutics’ LX2006 also holding Breakthrough Therapy, RMAT, Orphan Drug, and Fast Track designations. Solid Biosciences’ dual‑route approach offers a unique strategy to deliver frataxin to both the brain and heart, potentially improving efficacy and safety profiles compared to single‑route candidates.
"The Orphan Drug Designation and the first patient dose are milestones that validate our science and accelerate our path to patients," said Bo Cumbo, President and CEO. "The dual‑route delivery of SGT‑212 addresses the multisystem nature of Friedreich’s ataxia and positions us to meet the unmet needs of patients and families. We remain focused on advancing the FALCON trial and delivering meaningful outcomes for this rare disease.”
"The regulatory approvals and the first patient dose confirm that our preclinical work is translating into clinical progress," added Jessie Hanrahan, Ph.D., Chief Regulatory & Preclinical Operations Officer. "The Fast Track and Rare Pediatric Disease designations provide additional incentives and flexibility that will help us navigate the development pathway efficiently.”
"The first patient dose is a critical step in demonstrating safety and tolerability, and the early MRI imaging results are encouraging," said Gabriel Brooks, M.D., Chief Medical Officer. "We are optimistic about the potential of SGT‑212 to address the underlying cause of Friedreich’s ataxia and to improve patient outcomes.”
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