Passage Bio’s PBKR03 Receives Orphan Drug and Rare Pediatric Disease Designations from FDA for Treatment of Krabbe Disease

PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) — Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous systemThe central nervous system (CNS) is made up of the brain and the spinal cord. disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell LeukodystrophyAnother name for Krabbe disease, a rare and usually fatal disorder of the nervous system.). 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 diseaseLysosomal storage disorders comprise a group of ~50 metabolic disorders that result from a missing, deficient or lack of a specific enzyme. that presents early in the patient’s life, resulting in progressive damage to both the brain and peripheral nervous systemThe nervous system of the body is made up of the Central Nervous System (CNS) and the Peripheral Nervous System (PNS)..

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