FRIEDREICH'S ATAXIA RESEARCH ALLIANCE

Solid Biosciences' SGT-212 Receives FDA Fast Track Designation for Friedreich's Ataxia Treatment

5 months ago

• Solid Biosciences' SGT-212, a dual-route gene therapy for Friedreich's ataxia (FA), has received Fast Track designation from the FDA, expediting its development and review process. • SGT-212 delivers the frataxin gene via intravenous and direct intradentate nucleus infusions, targeting both neurological and cardiac manifestations of FA. • A Phase 1b clinical trial is planned for the second half of 2025 to assess the safety and tolerability of SGT-212 in adult FA patients. • The Fast Track designation will allow Solid Biosciences to have more frequent interactions with the FDA and the potential to be eligible for priority review.

Vatiquinone Shows Durable Benefit in Friedreich Ataxia, NDA Submission Planned

8 months ago

• PTC Therapeutics' vatiquinone demonstrated a 3.7-point benefit on the mFARS scale over 144 weeks, indicating a 50% slowing of Friedreich Ataxia progression. • Long-term data from an earlier study showed a 4.8-point mFARS benefit after 24 months of vatiquinone treatment compared to natural history. • Vatiquinone continues to exhibit a strong safety profile, with no treatment-related serious adverse events reported in long-term studies. • PTC Therapeutics plans to submit an NDA for vatiquinone to the FDA in December 2024, including data from the MOVE-FA trial and long-term extensions.