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

- SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations -

- Dosing of the first participant in the Phase 1b FALCON trial has been completed, with initial data expected in H2 2026, subject to participant enrollment -

CHARLESTOWN, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today 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). Additionally, earlier today, the Company reported that the first participant has been dosed in FALCON, a Phase 1b, first-in-human clinical trial evaluating SGT-212 for the treatment of FA.

“Receiving Orphan Drug, Fast Track and Rare Pediatric Disease designations underscores the significant unmet need the FA community faces and recognizes the therapeutic potential of SGT-212’s novel, dual-route administration,” said Jessie Hanrahan, Ph.D., Chief Regulatory & Preclinical Operations Officer of Solid Biosciences. “We anticipate that the ability to leverage these regulatory designations will help streamline and potentially accelerate SGT-212’s development path, and we look forward to working closely with regulators to bring a potential new treatment option to the FA community.”

SGT-212 is a first-in-class investigational gene therapy for the treatment of FA that employs a dual-route administration designed to target the neurologic, cardiac and systemic manifestations of the disease, which are key drivers of morbidity and mortality seen in FA. SGT-212 is delivered through a precise, stereotactic, MRI-guided intradentate nuclei (IDN) infusion to the cerebellar dentate nuclei followed by an intravenous (IV) infusion, with the goal of restoring therapeutic levels of frataxin.

About Orphan Drug Designation
The U.S. Food and Drug Administration (FDA) grants Orphan Drug designation (ODD) 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. ODD provides certain development incentives, including tax credits for qualified clinical testing, waiver of FDA application fees and seven years of market exclusivity upon approval. These benefits are designed to encourage innovation and to potentially accelerate the availability of treatments for patients with serious underserved conditions.