Development of a CoQ10 Headgroup Intermediate (4-HB) to Treat a Novel Pediatric Mitochondrial Encephalopathy

National Center For Advancing Translational Sciences

Investigators

Abstract

The formulation development for reducing the drug product dose volume of 4-HB administered to patients has begun using research-grade (non-GMP) drug substance. Efforts to manufacture GMP-compliant material will begin soon. Liquid chromatography assay development for quantification and impurity assessment of 4-HB has begun to facilitate formulation development and 4-HB solubility studies. Solubility in several excipients and blends have been examined, and further solubility studies of individual excipients and characterization of excipient blends are planned. Characterization of the final solution/suspension formulation and efforts to improve the formulation palatability will be undertaken. Once the formulation is selected, pilot batch production and stability studies will be completed.