Drugs

Saol Therapeutics Resubmits Rare Disease Therapy SL1009

Saol Therapeutics has resubmitted its NDA for SL1009 (sodium dichloroacetate, or DCA), an investigational treatment for pyruvate dehydrogenase complex deficiency (PDCD), a rare and life-threatening mitochondrial disorder with no currently approved therapies.

The resubmission follows additional analyses requested by FDA after the agency issued a Complete Response Letter (CRL) in 8/2025, according to the company. Saol says the CRL did not identify concerns related to the product’s safety profile or manufacturing, but instead requested additional evidence supporting the application.

When the company first received the CRL, it said it would “take several years and require significant financial resources” to address the deficiencies as FDA requested.” However, it says it was able to address FDA’s concerns through analyses of existing data, including survival outcomes, functional measures, mechanistic evidence, and long-term safety information, allowing it to resubmit the application without conducting an additional clinical trial.

Saol notes the path forward was established through discussions with FDA during a Type A meeting in December and a Type C meeting in March. During those meetings, the agency recommended additional survival analyses to support the NDA.

PDCD is described as a genetic disorder caused by defects in the pyruvate dehydrogenase complex, a critical cellular pathway involved in converting nutrients into energy. The disease can lead to chronic energy deficits, lactic acidosis, developmental impairment, neurological complications, and early childhood death.

SL1009 is designed to target the underlying metabolic dysfunction associated with the disease and would be administered with a proprietary dose-determining genetic test intended to identify patients most likely to benefit from treatment, according to the company.

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