Oral Pyridoxal 5′-Phosphate Fast Tracked for PNPO Deficiency

The Food and Drug Administration (FDA) has granted Fast Track designation to MC-1 for the treatment or prevention of seizures associated with pyridox(am)ine 5′-phosphate oxidase (PNPO) deficiency.

PNPO deficiency is a rare neurometabolic disorder caused by mutations in the PNPO gene, which is required for the production of normal levels of pyridoxal 5′-phosphate. The disorder is characterized by the onset of drug-resistant seizures in the first year of life. MC-1 is an investigational agent that contains pyridoxal 5′-phosphate monohydrate, an active vitamin B6 metabolite.

A phase 3, open-label study (MEND-PNPO; ClinicalTrials.gov Identifier: NCT04706013) evaluating MC-1 in patients with confirmed PNPO deficiency (via genetic analysis) is currently in the works, according to Medicure Inc. Patients will receive MC-1, a pharmaceutical grade pyridoxal 5′-phosphate, as an oral tablet.

The primary endpoint of the trial is overall survival time; the study group will be compared to a historical control group. Seizure frequency will also be assessed as a secondary outcome measure.

The FDA’s Fast Track designation helps to accelerate the development and review of products for serious and life-threatening conditions where no treatment exists or where the investigational therapy is likely to provide an advantage over currently available treatments.