Passage Bio’s (NASDAQ:PASG) PGBM01 received FDA rare pediatric disease designation for the treatment of GM1 gangliosidosis.
GM1 gangliosidosis is a rare genetic disease where mutations in the GLB1 gene impair production of beta-galactosidase, causing lipids to accumulate in cells of the nervous system.
PBGM01, an adeno-associated virus delivery gene therapy, has been previously granted FDA orphan drug designation.
“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,” Bruce Goldsmith, Passage Bio’s president and CEO, said in a statement.
“We believe that PBGM01 has the potential to restore developmental progression, enabling patients to achieve additional milestones and improve quality of life,” he added.
The company plans to begin a Phase 1/2 trial for PBGM01 for the treatment infantile GM1 gangliosidosis in the fourth quarter of 2020.