Synlogic Receives Positive Opinion on Orphan Designation from the European Medicines Agency for SYNB1934 for the Treatment of Phenylketonuria
“We are pleased that the EMA has issued a positive opinion for orphan designation for SYNB1934, in recognition of the urgent need for new treatment options for people living with this devastating disease,” said
SYNB1934 has also been granted Rare Pediatric Disease Designation by the
About the European Medicines Agency Orphan Designation
The EMA’s orphan designation is available to companies developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the European Union (EU). Medicines that meet the EMA’s orphan designation criteria qualify for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase and access to centralized marketing authorization.
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.