Synlogic, 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.
