pegtibatinase

Pegtibatinase is an investigational, PEGylated, recombinant enzyme replacement therapy designed to treat classical homocystinuria (HCU), a rare metabolic disorder caused by deficiency of the enzyme cystathionine beta synthase (CBS). Pegtibatinase is a genetically engineered form of the human CBS protein, modified for improved stability and activity in circulation. It works by metabolizing homocysteine to cystathionine in the bloodstream, thereby reducing toxic levels of homocysteine that can lead to serious complications affecting vision, skeletal system, circulatory system, and central nervous system. The drug is administered subcutaneously and has shown rapid and sustained reductions in total homocysteine levels in clinical studies. Pegtibatinase was originally developed by Orphan Technologies and further advanced by Travere Therapeutics (formerly Retrophin). It has received Breakthrough Therapy designation as well as Rare Pediatric Disease and Fast Track designations from the FDA, along with Orphan Drug status in both the US and Europe[1][3][5][6].

Other names

pegtibatinasePEGylated cystathionine beta-synthasePEG-CBS