This development marks the start of a second review cycle for the investigational drug, known chemically as sodium dichloroacetate. Saol Therapeutics previously received a Complete Response Letter from the agency in August 2025, though that notice did not cite issues regarding the drug's safety profile or manufacturing processes. Following subsequent consultations with federal reviewers in late 2025 and early 2026, the company provided the requested survival analyses to bolster its filing.
"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," said Dave Penake, chief executive officer of Saol Therapeutics. If approved, the drug will be administered alongside a companion genetic test developed with Medosome Biotec to determine appropriate dosing. The therapy currently holds Priority Review, Orphan Drug, and Rare Pediatric Disease designations, potentially positioning the company to receive a Priority Review Voucher upon successful authorization.
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