Synlogic Receives Orphan Drug Designation from FDA for SYNB1934 for Treatment of Phenylketonuria
“We are very pleased that SYNB1934 has been granted another regulatory designation, further validating the need for new treatment options for those living with PKU,” said
ODD is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, which generally affects less than 200,000 individuals in the
SYNB1934 has also received Rare Pediatric Disease Designation (RPDD) from the FDA and orphan designation from the
SYNB1934 is an orally administered, non-systemically absorbed drug candidate being studied as potential biotherapeutic for phenylketonuria (PKU). PKU is an inherited rare metabolic disease caused by an inborn error of metabolism that impairs the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. Treatment options for PKU are currently limited, with a majority of individuals with PKU in need of treatment or not adequately responding to treatment.
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Source: Synlogic, Inc.