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Reata’s Skyclarys Approval Aided By Natural History And Delayed-Start Analyses

 
• By Bridget Silverman

The first treatment for the rare neurodegenerative disorder Friedreich’s ataxia relied on a single placebo-controlled efficacy trial.

Frataxin
REDUCED EXPRESSION OF THE FRATAXIN MITOCHONDRIAL PROTEIN CAUSES FRIEDREICH'S ATAXIA. • Source: Shutterstock

Reata Pharmaceuticals, Inc. notched its first approval from its pipeline of drug candidates that activate the transcription factor Nrf2 when the US FDA approved Skyclarys (omaveloxolone) on 28 February 2023 as the first treatment for the Friedreich’s ataxia, an ultra-rare neurodegenerative disorder that is typically diagnosed in adolescence.

Reata emphasized clarity on FDA expectations for confirmatory evidence during the clinical development of omaveloxolone, which the company planned to submit with a single efficacy

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