PASG - FDA clears Passage Bio's application for PBKR03 in Krabbe disease

The FDA clears Passage Bio's (PASG) investigational new drug ((IND)) application for PBKR03, an adeno-associated virus ((AAV))-delivery gene therapy being studied for the treatment of early infantile Krabbe disease.Krabbe disease is a rare lysosomal storage disease that most often presents early in a child’s life, resulting in rapid progressive damage to both the brain and peripheral nervous system and mortality by two years of age. The company expects to initiate a Phase1/2 trial for PBKR03 in H1 2021. The primary endpoint is safety and tolerability; secondary endpoints include CSF and serum GALC levels, disease biomarkers, and clinical outcome measures.Initial data are are anticipated in late 2021 or early 2022.The FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 in Krabbe disease.

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FDA clears Passage Bio’s application for PBKR03 in Krabbe disease