FDA rejection leaves Saol searching for a path forward in rare pediatric disease

Saol Therapeutics has been dealt a blow in its effort to bring SL1009, an oral solution of sodium dichloroacetate (DCA), to patients with pyruvate dehydrogenase complex deficiency (PDCD). The US Food and Drug Administration (FDA) has issued a complete response letter rejecting the new drug application, despite the candidate holding orphan drug, priority review, and rare pediatric disease designations.

The regulator outlined deficiencies that Saol must address before the program can advance but did not cite any manufacturing concerns. According to the company, addressing the FDA’s requirements could take years and require resources beyond its current capacity. Even so, chief executive Dave Penake said Saol intends to request a Type A meeting with the FDA to discuss alternatives that would avoid another lengthy Phase III trial.