FDA Denies Approval of Dichloroacetate for Rare Mitochondrial Disorder

The Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Saol Therapeutics regarding the New Drug Application (NDA) for SL1009 (sodium dichloroacetate oral solution) for the treatment of pyruvate dehydrogenase complex deficiency (PDCD).

According to Saol, the CRL included specific requirements that “would take several years and require significant financial resources.” There are currently no FDA-approved treatments for PDCD, a mitochondrial disorder of carbohydrate oxidation that leads to decreased adenosine triphosphate production resulting in neurological and neuromuscular dysfunction.

The NDA for SL1009 in PDCD was supported by data from two phase 3 studies, a double-blind, placebo-controlled trial with an open-label extension phase (ClinicalTrials.gov Identifier: NCT02616484), and a survival analysis comparing treatment with a natural history cohort. The Company is currently seeking a path forward with the FDA that would not require an additional clinical trial.

“Traditional clinical trials often take many years; time that children with PDCD simply do not have,” said Dave Penake, CEO of Saol Therapeutics. “We remain committed to productive conversations with the Agency to identify a path forward that brings this therapy to patients as swiftly as possible.”

Access to SL1009 will continue through both the open-label extension portion of the phase 3 trial, as well the Expanded Access Program (ClinicalTrials.gov Identifier: NCT06931262).

This article originally appeared on MPR