Synlogic Receives Positive Opinion on Orphan Designation From the EMA for SYNB1618 for the Treatment of Phenylketonuria

Synlogic, Inc. recently announced the European Medicines Agency (EMA) issued a positive opinion on the company’s application for orphan designation for SYNB1618 for the treatment of phenylketonuria (PKU). Previously, the US FDA granted SYNB1618 both Orphan Drug Designation and Fast Track designation.

“We are pleased that the EMA has issued a positive opinion for orphan designation for SYNB1618, in recognition of the urgent need for additional treatment options for PKU,” said Aoife Brennan, MB, ChB, Synlogic President and Chief Executive Officer. “This designation reflects our constructive engagement with multiple regulatory agencies as we progress our PKU program into late-stage development. We look forward to advancing our PKU program into a registrational trial in the first half of 2023.”

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.

SYNB1618 and SYNB1934 are orally administered, non-systemically absorbed drug candidates being studied as potential biotherapeutics for phenylketonuria (PKU), an inherited metabolic disease marked by an inability to break down the amino acid phenylalanine (Phe), which can be neurotoxic. 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 its drug candidates 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. SYNB1618 has received both Orphan Drug and Fast Track designations by the US Food and Drug Administration (FDA) and a positive opinion on Orphan Designation from the European Medicines Agency (EMA).

Synlogic is a clinical-stage biotechnology company developing medicines through its proprietary approach to synthetic biology. Synlogic’s pipeline includes its lead program in phenylketonuria (PKU), which has demonstrated proof of concept with plans to start a pivotal, Phase 3 study in the first half of 2023, and additional novel drug candidates designed to treat homocystinuria (HCU) and enteric hyperoxaluria. The rapid advancement of these potential biotherapeutics, called Synthetic Biotics, has been enabled by Synlogic’s reproducible, target-specific drug design. Synlogic uses programmable, precision genetic engineering of well-characterized probiotics to exert localized activity for therapeutic benefit, with a focus on metabolic and immunologic diseases. In addition to its clinical programs, Synlogic has research collaborations with Roche on the discovery of a novel Synthetic Biotic for the treatment of inflammatory bowel disease and with Ginkgo Bioworks on additional undisclosed preclinical assets. For more information, visit www.synlogictx.com.