Synlogic Receives Orphan Drug Designation from FDA for SYNB1934 for Treatment of Phenylketonuria
CAMBRIDGE, Mass., May 09, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to SYNB1934 for the treatment of phenylketonuria (PKU).
“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 Aoife Brennan M.B. Ch.B., Synlogic President and Chief Executive Officer. “This designation also comes at a pivotal time as we prepare to initiate our Phase 3 trial for PKU – Synpheny-3— in the first half of this year.”
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 U.S. ODD granted therapies entitle companies to development incentives including tax credits for qualified clinical trials, user fee exemptions, and the potential for seven years of market exclusivity after approval.
SYNB1934 has also received Rare Pediatric Disease Designation (RPDD) from the FDA and orphan designation from the European Medicines Agency (EMA).
About SYNB1934
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. Synlogic designed SYNB1934 to reduce levels of Phe in people with PKU by consuming Phe in the gastrointestinal (GI) tract, using genetic engineering of the well-characterized probiotic E. coli Nissle. Findings to date support the potential for an oral, efficacious, safe, convenient, and flexible treatment option for PKU. SYNB1934 has been granted Rare Pediatric Disease and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and orphan designation from the European Medicines Agency (EMA).
About Synlogic
Synlogic is the leading company advancing therapeutics based on synthetic biology. Synlogic’s pipeline includ
