Synlogic Granted Fast Track Designation from FDA for labafenogene marselecobac (SYNB1934) for Treatment of Phenylketonuria

CAMBRIDGE, Mass., July 11, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company advancing novel, oral, non-systemically absorbed biotherapeutics to transform the care of serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to labafenogene marselecobac (previously known as SYNB1934) for the treatment of phenylketonuria (PKU). Labafenogene marselecobac has also received Rare Disease Designation (RPDD) and Orphan Drug Designation (ODD) by the FDA and orphan designation from the European Medicines Agency (EMA).

“We are pleased that this potentially transformative therapy has now received three important regulatory designations from the FDA, and orphan designation from the EMA, reflecting a shared understanding of the urgent need for new medical treatment options that can effectively and safely lower Phe levels in patients with PKU,” said Aoife Brennan, M.B. Ch.B., Synlogic President and Chief Executive Officer. “This milestone re-enforces our own urgency as we execute our pivotal study, Synpheny-3.”

The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. To qualify, available clinical and non-clinical data need to demonstrate meaningful therapeutic potential. The benefits of Fast Track designation include opportunities for frequent meetings with the FDA to discuss trial design, development plans and data needed to support drug approval, as well as the ability to submit a registrational filing for approval on a rolling basis, and eligibility for priority review, if relevant criteria are met.

About labafenogene marselecobac

Labafenogene marselecobac (previously known as SYNB1934) is an orally administered, non-systemically absorbed, potential treatment for phenylketonuria (PKU), a rare metabolic disease caused by inherited mutations that impair the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. The goal of PKU management is to reduce plasma Phe below neurotoxic levels, reducing risk of neurocognitive complications. Current treatment options for PKU are limited due to safety and efficacy, leaving the majority of people living with PKU without medical management and with uncontrolled Phe. Synlogic designed labafenogene marselecobac to target and consume Phe in the GI tract, by applying precision genetic engineering to a well-characterized probiotic. Results to date indicate the potential for labafenogene marselecobac as the first therapeutic for PKU approved as both a monotherapy and adjunctive medical treatment, and following successful Phase 2 results, it has advanced to Synpheny-3, a global, pivotal Phase 3 study. Labafenogene marselecobac has received Orphan Drug Designation (ODD), Fast Track designation and Rare Pediatric Disease Designation (RPDD) from the FDA in addition to orphan designation from the European Medicines Agency (EMA).