Saol Therapeutics Announces Submission of New Drug Application (NDA) to the U.S. FDA for SL1009
Wednesday, December 04, 2024
Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of SL1009, Sodium Dichloroacetate Oral Solution (DCA) for use with a proprietary genetic test, for the treatment of an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD)1. 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.
"This NDA submission is an important step in bringing this therapy to children living with PDCD in the U.S., where no approved treatment is currently available," said Dave Penake, Chief Executive Officer of Saol Therapeutics. "We look forward to continuing our ongoing discussions with the FDA as we move closer to addressing this significant unmet need of children suffering from 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). The totality of evidence submitted in the NDA includes mechanistic characterization, nonclinical and clinical safety and efficacy evidence to support the clinical benefit of DCA in PDCD patients.
The primary endpoint of SL1009-01 was a daily Observer Reported Outcomes survey tool to measure changes in motor domains (ObsROmotor) as well as the safety and tolerability of SL1009 compared to placebo. A key secondary endpoint measured the reduction in plasma lactate of SL1009 compared to placebo. Patients receiving DCA were dose-stratified post-randomization utilizing a proprietary genetic test. The test identifies each patient's GSTZ1 genotype to provide individualized dosing intended to reduce adverse events, such as peripheral neuropathy.
A second study, SL1009-02, was a survival analysis that compared outcomes for DCA-treated patients in the Phase III study with external, untreated natural history cohort of patients with PDCD. Patients were matched on age and sex.
Peter Stacpoole, MD, initial study sponsor of SL1009-01 said "The NDA submission is a tremendous milestone and the culmination of years of effort. I am grateful for the clinicians and families who participated in the trial."
Detailed data is expected to be presented at an upcoming medical conference.
DCA is not currently approved for any indication in the United States.
Source: prnewswire.com
