FDA Accepts Saol's Resubmitted NDA for SL1009 in Ultra-Rare Mitochondrial Disease
On July 28, 2026, Saol Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted its resubmitted New Drug Application (NDA) for SL1009 (sodium dichloroacetate / DCA) for the treatment of Pyruvate Dehydrogenase Complex Deficiency (PDCD), an ultra-rare and life-threatening mitochondrial disease with no FDA-approved therapies.
The FDA acknowledged the resubmission as a complete, Class 2 response and established a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2026.
Regulatory Context and Survival Analyses
This resubmission enters SL1009 into its second FDA review cycle. The original NDA was submitted on November 27, 2024, and subsequently received a Complete Response Letter (CRL) on August 27, 2025. Crucially, the CRL did not identify any safety or manufacturing concerns. Following a productive Type A meeting in December 2025 and a Type C meeting in March 2026, the FDA advised Saol to conduct and submit additional survival analyses to support the application, which Saol has now provided.1
If approved, Saol anticipates receiving a Priority Review Voucher (PRV) under the Rare Pediatric Disease Statute. Saol is also collaborating with Medosome Biotec to file a 510(k) application for a proprietary companion diagnostic—a dose-determining genetic test required to treat patients with SL1009.
Verbatim Quotes
Dave Penake, Chief Executive Officer of Saol Therapeutics, highlighted the significance of the target action date for the patient community:
"Having a confirmed action date matters greatly to the families of children living with PDCD, who have waited a long time for a potential treatment option. We remain confident in the totality of evidence behind SL1009 and what it can mean for these patients."
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An instance of Rare disease pipelines only accelerate through early FDA alignment and breakthrough designations. — Saol accelerated the re-evaluation of its rare disease candidate by actively consulting and aligning with the FDA on the necessary survival analyses to overcome a prior rejection. ↩︎