Labafenogene marselecobac is an oral non-systemically absorbed therapy that can act on and consume phenylalanine (Phe) in the gastrointestinal tract.
Data is currently showing the therapy’s ability to treat PKU both as a monotherapy and as an adjunctive treatment.
Labafenogene marselecobac completed the Phase II trial and is being progressed in the international Phase III Synpheny-3 trial.
The FDA also granted orphan drug designation (ODD) and rare paediatric disease designation for the therapy, which previously obtained ODD from the European Medicines Agency (EMA).
Fast track status from the FDA aids in developing and speeding up the review of therapies for serious conditions and for meeting an unmet medical need.
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PKU is a rare metabolic ailment caused by inherited mutations that impact Phe disintegration.
Synlogic president and CEO Aoife Brennan stated: “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.
“This milestone re-enforces our own urgency as we execute our pivotal study, Synpheny-3.”