Regulatory & Policy

Saol Therapeutics resubmits rare mitochondrial disease drug after FDA agreement

Roswell, Georgia-based Saol Therapeutics has resubmitted its New Drug Application (NDA) to the US FDA for SL1009 (sodium dichloroacetate, or DCA), seeking...

Edited by Robert McTiernan··2 min read
Saol Therapeutics resubmits rare mitochondrial disease drug after FDA agreement

Saol Therapeutics has resubmitted its New Drug Application (NDA) to the US FDA for SL1009 (sodium dichloroacetate, DCA) for the treatment of pyruvate dehydrogenase complex deficiency (PDCD), a rare, often fatal mitochondrial disease with no approved therapies. The resubmission follows a Complete Response Letter (CRL) issued in August 2025 and incorporates additional analyses requested by the FDA without requiring a new clinical trial.

Following the CRL, Saol held Type A and Type C meetings with the FDA, which recommended supplementary survival analyses using existing clinical data to strengthen the benefit-risk assessment. The company said this enabled it to proceed directly to resubmission rather than initiating another pivotal study.

PDCD is caused by defects in the pyruvate dehydrogenase complex, leading to impaired energy production, chronic lactic acidosis, severe neurological impairment, and high mortality in early-onset disease. Treatment is currently limited to ketogenic diets, nutritional supplementation, and supportive care.

SL1009 is an oral DCA solution intended for use with a companion genetic diagnostic developed with Medosome Biotec to identify GSTZ1 genotypes and guide individualized dosing, reducing the risk of peripheral neuropathy. The therapy has received FDA Orphan Drug, Rare Pediatric Disease, and Priority Review designations. If approved, Saol also expects to receive a Rare Pediatric Disease Priority Review Voucher.

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The NDA is supported by two Phase III studies and long-term extension data. While the primary endpoint of the first Phase III trial was not met during the blinded phase, patients receiving long-term treatment demonstrated significant improvements in motor function and reductions in plasma lactate. A second study comparing DCA-treated patients with a matched natural history cohort showed improved survival, and these analyses have been expanded in the resubmission.

If approved, SL1009 would become the first FDA-approved treatment for PDCD, providing the first pharmacological option for a disease currently managed only with supportive care. Access to the therapy has continued through an ongoing open-label extension study and expanded access program.

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