The investigational gene therapy for Friedreich’s ataxia (FA), SGT-212, has received Rare Pediatric Disease (RPD) Designation from the U.S. Food and Drug Administration (FDA), marking a significant milestone alongside its previously granted Fast Track designation, according to an announcement by Solid Biosciences.

FA is a severe, inherited, life-threatening, degenerative multisystem disease caused by defects in the frataxin (FXN) gene, impacting approximately 5,000 people in the United States, and currently lacking any treatments that halt disease progression. FA is a severe, inherited, life-threatening, degenerative multisystem disease caused by defects in the frataxin (FXN) gene, impacting approximately 5,000 people in the United States, and currently lacking any treatments that halt disease progression.

SGT-212 is the only dual-route gene therapy in development for FA, designed to deliver the full-length human frataxin gene via a unique dual route of administration: a direct intradentate nucleus (IDN) infusion followed by an intravenous (IV) infusion.

This approach aims to restore therapeutic frataxin protein levels, specifically targeting the cerebellar dentate nuclei and cardiomyocytes to address the disease's profound neurologic, cardiac, and systemic manifestations. The RPD designation, intended for serious pediatric conditions, offers the valuable incentive of potentially receiving a Pediatric Priority Review Voucher (PRV) upon product approval, a redeemable or transferable voucher that can be used to expedite the review of a future marketing application.

With these designations in place, Solid Biosciences is currently screening participants for the first-in-human trial, the FALCON Phase 1b clinical trial, as it works to accelerate the development and potential market entry of SGT-212.