Business
Passage Bio Receives Rare Pediatric Disease Designation for PBGM01 for Patients with GM1 Gangliosidosis
– PBGM01 to enter the clinic in fourth quarter of 2020 – PHILADELPHIA, May 21, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines
About this update from Passage Bio, Inc.
[{"type":"text","content":"– PBGM01 to enter the clinic in fourth quarter of 2020 –\nPHILADELPHIA, May 21, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to PBGM01 broadly for the treatment of GM1 gangliosidosis. PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis (GM1) and has previously been granted Orphan Drug designation.\n “This is the second regulatory designation we have received from the FDA for our lead program in GM1 and reflects the high unmet need in this patient population,” said Bruce Goldsmith, Ph.D, president and chief executive officer of Passage Bio. “As a company we are committed to developing therapies that transform the lives of patients suffering from serious life-threatening CNS disorders. We believe that PBGM01 has the potential to restore developmental progression, enabling patients to achieve additional milestones and improve quality of life. We look forward to advancing PBGM01 into clinical testing later this year.” RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. Under the RPD program, a sponsor who receives approval for a drug or biologic for a “rare pediatric disease” may qualify for a priority review voucher that may be sold or transferred. Passage anticipates starting a Phase 1/2 trial for PBGM01 for the treatment infantile GM1 in the fourth quarter of 2020. The trial will be an open-label, dose escalation study of PBGM01 administered by a single injection into the intra cisterna magna, or ICM, in pediatric subjects with infantile GM1. About GM1GM1 gangliosidosis (GM1) is a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid beta-galactosidase (β-gal). Reduced β-gal activity results in the accumulation of toxic levels of GM1 ganglioside in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is ...