Vatiquinone

Unassigned

New Medicines

Refractory seizures in children with inherited mitochondrial disease (mitochondrial epilepsy)

Information

New molecular entity
PTC Therapeutics
PTC Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

15-Lipoxygenase inhibitor (enzyme that regulates oxidative stress and inflammation response pathways)
Inherited mitochondrial epilepsy is a manifestation of mitochondrial disease estimated to affect approximately 11,000-13,000 children in the US, Europe, Japan and Latin America [1].
Refractory seizures in children with inherited mitochondrial disease (mitochondrial epilepsy)
Oral