ROSWELL, Ga., DUBLIN, Ireland & HAMILTON, Bermuda, July 7, 2026
Saol Therapeutics has announced the resubmission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for SL1009 (sodium dichloroacetate, DCA), an investigational therapy for Pyruvate Dehydrogenase Complex Deficiency (PDCD), an ultra-rare, life-threatening mitochondrial disease with no FDA-approved treatment options. The resubmission follows constructive discussions with the FDA through both Type A and Type C meetings, during which the Agency recommended additional survival analyses based on existing clinical data rather than requiring a new clinical trial. This regulatory pathway allowed Saol to strengthen its application using comprehensive evidence from previous studies, reinforcing the company’s commitment to accelerating access to innovative therapies for patients with serious rare diseases. The milestone represents a significant advancement for the PDCD community and reflects ongoing collaboration between regulators and developers working to address critical unmet medical needs in rare pediatric disorders.
FDA Engagement Supports Direct NDA Resubmission
The NDA resubmission follows the Complete Response Letter (CRL) received by Saol in August 2025, which requested additional evidence supporting the therapy’s clinical benefit but did not identify concerns regarding safety or manufacturing. Working closely with the FDA, Saol conducted additional analyses of existing datasets, including overall survival, functional outcomes, long-term safety data, and mechanistic evidence, allowing the company to proceed directly with resubmission without initiating another clinical trial.
According to Dave Penake, Chief Executive Officer of Saol Therapeutics, the constructive dialogue with the FDA demonstrates regulatory flexibility for therapies addressing diseases with substantial unmet medical need. The company believes the comprehensive body of evidence now submitted supports the potential clinical benefit of SL1009 and positions the application for regulatory review under an efficient pathway focused on patients with rare, life-threatening conditions.
SL1009 Targets an Ultra-Rare Mitochondrial Disease
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SL1009 (sodium dichloroacetate) is being developed as a precision therapy for Pyruvate Dehydrogenase Complex Deficiency (PDCD), a rare inherited mitochondrial disorder that causes severe energy metabolism dysfunction, chronic lactic acidosis, developmental impairment, neurological complications, and frequently early childhood death. The investigational therapy has been evaluated across multiple clinical studies, including two Phase 3 clinical trials and extensive long-term open-label extension studies, establishing a well-characterized safety profile while generating evidence supporting potential clinical benefit. If approved, SL1009 will be prescribed together with a proprietary companion genetic test designed to determine appropriate patient dosing.
The therapy has already received FDA Priority Review, Orphan Drug Designation, and Rare Pediatric Disease Designation, highlighting its importance for patients with limited treatment options. Saol also anticipates eligibility for a Priority Review Voucher (PRV) under the Rare Pediatric Disease Program if the therapy receives regulatory approval. The FDA is expected to assign a new Prescription Drug User Fee Act (PDUFA) action date following acceptance of the resubmitted application. With no approved therapies currently available for PDCD, the successful review of SL1009 could represent a major breakthrough for mitochondrial medicine, offering a potentially transformative treatment option for children and families affected by this devastating ultra-rare disease while reinforcing continued innovation in orphan drug development.
Source: Saol Therapeutics press release
