for pyruvate dehydrogenase complex deficiency (PDCD)
Originally MAY 7 2027; extended to DEC 30 2026 (FDA-assigned PDUFA replaced projected +10-month math. Resubmission classified as Class 2 (5-month clock)., announced JUL 28 2026).
Pyruvate dehydrogenase complex deficiency is a severe metabolic disorder that causes a harmful buildup of lactic acid in children, often leading to fatal neurological damage. Sodium dichloroacetate is an oral drug that works by blocking a specific enzyme, which helps the body's cellular power plants produce energy more normally. This mechanism may reduce the toxic acid levels and potentially improve neurological outcomes for affected children.
The objective of this research study is to conduct a pivotal phase 3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital lactic acidosis and is a frequently fatal metabolic disease of childhood for which no proven treatment exists
Source: ClinicalTrials.gov