bitopertin

Bitopertin is a first-in-class, oral small molecule inhibitor of glycine transporter 1 (GlyT1). By inhibiting GlyT1, it increases synaptic glycine levels and modulates heme biosynthesis by limiting glycine uptake. This mechanism was originally explored as adjunctive therapy for schizophrenia to enhance NMDA receptor function but failed in late-stage trials for that indication. Bitopertin is now in advanced clinical development for erythropoietic protoporphyria (EPP) and other erythropoietic porphyrias, where it aims to reduce the accumulation of protoporphyrin IX by restricting heme pathway metabolite production. The drug has received orphan drug designation from the FDA for EPP and is currently being evaluated in phase 2/3 studies. It was initially developed by Roche and is now being developed by Disc Medicine[1][2][4][7].

Other names

bitopertinumPaliflutine