PTC Therapeutics (PTCT) Q1 2025 Earnings Call

May 6, 2025. Vatiquinone (Friedreich's Ataxia): FDA review proceeding without AdCom; confirmatory evidence from long-term data compared to FA-COMS natural history registry showing a reported 50% slowing in disease progression over three years.

Omaveloxolone for treating Friedreich’s ataxia in people 16 years and over (terminated appraisal)

National Institute for Health and Care Excellence (NICE). 06 May 2025. 

NICE is unable to make a recommendation on omaveloxolone (Skyclarys) for treating Friedreich’s ataxia in people 16 years and over. This is because Biogen withdrew its evidence submission.