SL1009 FDA Approval Status
Last updated by Judith Stewart, BPharm on Jan 20, 2025.
FDA Approved: No
Brand name: SL1009
Generic name: sodium dichloroacetate
Dosage form: Oral Solution
Company: Saol Therapeutics
Treatment for: Pyruvate Dehydrogenase Complex Deficiency
SL1009 (sodium dichloroacetate) is a pan- pyruvate dehydrogenase kinase (PDK) inhibitor in development for the treatment of Pyruvate Dehydrogenase Complex Deficiency.
- Pyruvate Dehydrogenase Complex Deficiency (PDCD) is a rare disorder of carbohydrate oxidation caused by gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC). PDCD mostly affects the nervous system and skeletal muscle, leading to decreased adenosine triphosphate (ATP) production and energy failure. Symptoms include lactic acidosis, lethargy, poor feeding, rapid breathing, and other signs of neurological and neuromuscular dysfunction such as developmental delay, hypotonia, abnormal eye movements and seizures.
- SL1009 works by inhibiting pyruvate dehydrogenase kinases (PDK) to stimulate residual PDC activity and increase ATP production by the mitochondria. Individualized dosing is based on the results of a GSTZ1/MAAI genetic test that dichotomizes subjects into "fast" and "slow" drug metabolizers.
- A New Drug Application (NDA) has been submitted to the U.S. Food and Drug Administration (FDA) for SL1009 for use with a genetic test for the treatment of PDCD. The NDA is supported by results from the Phase 3 double-blind placebo controlled cross-over study (SL1009-01) and a survival study (SL1009-02) which support the clinical benefit of DCA in PDCD patients.
- SL1009 has been granted Orphan Drug, Fast Track and Rare Pediatric Disease Designations by the FDA and is thus eligible for Priority Review and a Priority Review Voucher.
Development timeline for SL1009
Further information
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