Saol Positions Ultrarare PDCD Therapy SL1009 as Test Case for FDA's New Rare Disease Evidence Principles
SHERIDAN, WYOMING - December 11, 2025 - Saol Therapeutics is turning an FDA rejection into a potential regulatory test case, as the biotech prepares to argue that its pyruvate dehydrogenase complex deficiency (PDCD) drug SL1009 is the ideal "poster child" for the agency's new Rare Disease Evidence Principles (RDEP) framework at a Type A meeting on December 18.
Saol Recasts a Complete Response Letter as a Strategic Opening
On September 8, Saol disclosed that the FDA had issued a complete response letter (CRL) for SL1009, an oral formulation of sodium dichloroacetate for children with PDCD, a genetic disorder affecting fewer than 1,000 people in the U.S. The CRL, according to CEO Dave Penake, "suggested that we would need to do an additional adequate and well controlled clinical trial," and that "and that's not feasible to be done by our company and in this patient population."