Synlogic receives orphan drug designation for SYNB1618.
Synlogic announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to SYNB1618, Synlogic's preclinical-stage drug candidate for the treatment of phenylketonuria , an inborn error of metabolism caused by a mutation in the gene that breaks down the amino acid phenylalanine. Phe accumulation in the blood and brain can lead to neurocognitive abnormalities and treatment currently requires severe dietary protein restriction. SYNB1618, an orally administered medicine, is designed to complement the missing function in patients with PKU by providing alternative metabolic pathways to consume Phe. Synlogic plans to file an investigational new drug application with the FDA for SYNB1618 for the potential treatment of PKU in early 2018.
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|Date:||Oct 24, 2017|
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