Posted by Michael Wonder on 29 Oct 2020
Passage Bio’s PBKR03 receives orphan drug and rare paediatric disease designations from FDA for treatment of Krabbe disease
28 October 2020 - Passage Bio today announced that the U.S. FDA has granted orphan drug and rare paediatric 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.
