Solid Biosciences’ SGT-212 for Friedreich’s Ataxia Gets FDA Fast Track

Solid Biosciences Inc., a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that it has received Fast Track designation from the US Food and Drug Administration (FDA) for SGT-212, the company’s, AAV-based gene therapy candidate for the treatment of Friedreich’s ataxia (FA). SGT-212 will deliver the full-length frataxin gene via dual routes of administration incorporating intradentate nucleus (IDN) and intravenous (IV) infusions, designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA.

Fast Track designation is granted to products that are developed to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. This designation is intended to facilitate development and expedite review of qualifying drugs. SGT-212 will benefit from this designation by having more frequent interactions with the FDA and the potential to be eligible for priority review.

Bo Cumbo, president and CEO commented: “SGT-212 is the only FA therapy in development that is designed to address frataxin deficiency, the underlying cause of FA, and all manifestations of this devastating disease, and in doing so, hopefully halt the full spectrum of symptom progression regardless of where the patient is in their journey with this terrible disease. We believe Fast Track designation may enable us to more rapidly develop SGT-212 and bring hope to those living with FA who need and deserve better treatment options.”

FDA IND clearance for SGT-212 was announced January 7, 2025. The planned phase 1b trial will be a first-in-human, open-label, multicenter study to evaluate the safety and tolerability of contemporaneous systemic IV and bilateral IDN administration of SGT-212 in adult non-ambulatory and ambulatory patients with FA. Dosing is expected to initiate in the second half of 2025 and participants in the trial will be followed for five years after receiving SGT-212.

“We are grateful for the FDA’s recognition that the needs of the FA community remain underserved, and that SGT-212 has the potential to bring meaningful change to their lives,” said Jessie Hanrahan, Ph.D., chief regulatory officer at Solid Biosciences. “We look forward to working closely with the Agency to discuss the most effective and expeditious development pathway for SGT-212 to pursue future marketing authorization.”

The FDA’s Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich’s ataxia (FA) designed to deliver full-length human frataxin (FXN) via a dual route of administration: intradentate nucleus (IDN) infusion, using an FDA-approved, stereotactic, precision MRI-guided device, followed by an intravenous (IV) infusion to increase therapeutic FXN levels in the cerebellar dentate nuclei and in the cardiomyocytes, respectively. Targeted delivery to the dentate nuclei will be confirmed in real time via gadolinium, an MRI-enhancing contrast agent. Restoration of FXN levels is expected to repair the underlying mitochondrial dysfunction in neurons and cardiomyocytes to address neurologic, cardiac and systemic manifestations of the disease.

FA is an inherited, life-threatening, degenerative multisystem disease caused by defects in the frataxin gene that disrupt production of the frataxin protein, a mitochondrial iron-binding protein involved in essential cellular processes, including energy production. FA is known to cause progressive nervous system damage, movement problems, and cardiac dysfunction, with cardiac complications identified as the primary cause of death. FA impacts approximately 5,000 people in the United States and 15,000 in Europe. There are currently no treatments that provide a cure or halt disease progression.

Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (Duchenne), Friedreich’s ataxia (FA), catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy, BAG3-mediated dilated cardiomyopathy, and additional fatal, genetic cardiac diseases.

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