FDA Grants Passage Bio Orphan Drug Designation for PBGM01 for Treatment of Infantile GM1 Gangliosidosis

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[{"type":"text","content":"PHILADELPHIA, April 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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead product candidate, PBGM01, for the treatment of infantile GM1 gangliosidosis (GM1). GM1 is a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene that results in rapidly progressing neurodegeneration.\n “Infantile GM1 is an incredibly devastating disease that impacts one of our most vulnerable populations,” said Bruce Goldsmith, Ph.D, chief executive officer of Passage Bio. “This designation represents an important recognition of the dire need for an effective treatment option for the children and their families impacted by GM1. We believe PBGM01 has the potential to be life-altering, and we look forward to bringing PBGM01 into clinical development later this year and to continuing to work with the FDA as we advance this therapy through development and generate clinical data in 2021.” The FDA’s Office of Orphan Products Development (OOPD) grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation qualifies recipients to certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval. About GM1 GM1 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 most severe in the Infantile form, which is characterized by onset in the first 6 months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, a...

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