Vatiquinone is a first-in-class small-molecule selective 15-lipoxygenase inhibitor developed for Friedreich's ataxia. In August 2025, FDA issued a Complete Response Letter for the NDA and did not approve vatiquinone, requesting an additional adequate and well-controlled study. It therefore remains investigational.
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07159139 |
Vatiquinone Expanded Access Protocol |
NO_LONGER_AVAILABLE | |
| NCT05515536 |
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05485987 |
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia |
COMPLETED | PHASE2 |
| NCT05218655 |
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease |
COMPLETED | PHASE3 |
| NCT04577352 |
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia |
COMPLETED | PHASE2/PHASE3 |
| NCT04378075 |
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy |
TERMINATED | PHASE2/PHASE3 |
