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Passage Bio Receives European Commission Orphan Designation for PBKR03 for Treatment of Krabbe Disease
- Krabbe disease, a rare lysosomal storage disorder, has no approved disease-modifying treatment options - Urgency for effective treatment underscored by
About this update from Passage Bio, Inc.
[{"type":"text","content":"- Krabbe disease, a rare lysosomal storage disorder, has no approved disease-modifying treatment options - Urgency for effective treatment underscored by European Commission designation for investigational gene therapy PBKR03 - Global Phase 1/2 trial – GALax-C – PBKR03 planned to initiate in first half of 2021 PHILADELPHIA, April 05, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the European Commission has granted Orphan designation for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). 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 disease 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. “We are planning to initiate in the first half of this year our clinical trial program, GALax-C, to study PBKR03 as a treatment for early infantile Krabbe disease, the most common and severe form of the disorder,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “We're pleased to receive Orphan designation from the European Commission, which — along with multiple FDA designations — reinforces the potential of our approach and the urgent unmet medical need for treatments for infants afflicted with this devastating disease.” PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C). Low GAL-C activity results in accumulation of psychosine, which is toxic to the myelin-producing oligodendrocytes of the CNS and Schwann cells in the periphery, resulting in damage to both the central and peripheral nervous systems. PBKR03 has the potential to treat both the central nervous system and peripheral nerve manifestations observed in K...