Passage Bio, Inc., a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, announced that the FDA has granted Orphan Drug and Rare Pediatric Disease designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy).
Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021. Krabbe disease is a rare and often life-threatening lysosomal storage disease that presents early in the patient’s life, resulting in progressive damage to both the brain and peripheral nervous system.