Identified literature showed a considerable unmet need for therapeutic interventions that halt or slow the deteriorating nature of FA. Novel efficacious drugs should be investigated that aim to improve symptoms or slow disease progression.
Thursday, December 1, 2022
Clinical evidence of interventions assessed in Friedreich ataxia: a systematic review
Jain P, Badgujar L, Spoorendonk J, Buesch K.; Therapeutic Advances in Rare Disease. 2022;3. doi:10.1177/26330040221139872
Friedreich’s ataxia: major trial readouts and regulatory events to watch in 2023
www.clinicaltrialsarena.com; Analysis. November 30, 2022
The FDA could approve the first treatment for Friedreich’s ataxia, and at least three other major trials have readouts expected by year end.
All signs indicate 2023 will prove a pivotal year in Friedreich’s ataxia drug development. Reata’s omaveloxolone, which met its primary endpoint in a Phase II trial, is up for FDA approval on February 28. Meanwhile, a Phase II/III trial of PTC’s vatiquinone, a Phase I/II trial of Stealth Bio’s elamipretide, and a Phase II study of Larimar’s CI-1601 all have key results expected in 2023.
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