FDA Returns Complete Response Letter for Saol’s Rare Genetic Disorder Treatment

Saol Therapeutics received an FDA complete response letter (CRL) for its NDA on sodium dichloroacetate (SL-1009), an experimental drug for pyruvate dehydrogenase complex deficiency, a rare pediatric mitochondrial disorder. The FDA’s rejection underscores the challenges of developing therapies for ultra-rare diseases with small patient populations. Saol’s inhibitor of pyruvate dehydrogenase kinases was the sole candidate in development for this condition. The CRL has sparked advocacy groups to urge increased policy focus and support for treatment development in rare genetic diseases.