Passage Bio Receives European Commission Orphan Designation for PBKR03 for Treatment of Krabbe Disease

PHILADELPHIA, April 5, 2021 (GLOBE NEWSWIRE) —  Passage Bio, Inc., (Nasdaq: PASG), a clinical-stage 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. (CNS) disorders, today announced that the European Commission has granted Orphan designation for PBKR03, an adeno-associated virus (AAV)-delivery gene therapyA type of therapy that offers hope and promise for a cure for many genetic disorders.  A working copy of the gene replaces the non-working copy of the gene. Gene therapy is at the forefront of many for the treatment of Krabbe disease (Globoid Cell LeukodystrophyAnother name for Krabbe disease, a rare and usually fatal disorder of the nervous system.). The designation was based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare 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 most often presents early in a child’s life. The U.S. Food and Drug Administration (FDA) previously granted Fast Track, Orphan Drug and Rare Pediatric Disease designations to PBKR03 for the treatment of Krabbe disease.

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