What is pegtibatinase?
Pegtibatinase, also known as TVT-058, is an investigational PEGylated recombinant enzyme replacement therapy being developed by Travere Therapeutics for people with classical homocystinuria (HCU) caused by cystathionine beta synthase (CBS) deficiency.
Classical HCU occurs when CBS enzyme activity is deficient. This disrupts the normal metabolism of homocysteine and can result in elevated total homocysteine levels.
Pegtibatinase is designed to replace deficient CBS activity, providing another approach to reducing elevated homocysteine. It remains an investigational therapy and has not been approved by a regulatory authority.
From COMPOSE to HARMONY
Pegtibatinase was initially evaluated through the COMPOSE Phase 1/2 clinical development program. Travere reported dose-dependent reductions in total homocysteine during the program, with pegtibatinase generally well tolerated across the doses studied.
The research program then advanced to HARMONY, a global, randomized, multi-centre, double-blind and placebo-controlled Phase 3 study. HARMONY is evaluating the efficacy and safety of pegtibatinase when administered in addition to standard-of-care treatment for classical HCU.
A related study, ENSEMBLE, was established as an open-label long-term extension to evaluate the ongoing efficacy and longer-term safety of pegtibatinase in eligible participants completing earlier studies in the program.
Clinical trial status, participating locations, eligibility requirements and study timelines can change. For that reason, CanPKU+ recommends using the official study and sponsor links below for current information rather than relying on this article for recruitment decisions.
For Canadians interested in this research, the most useful next step is to check current clinical trial sources for Canadian locations, recruitment status and eligibility information. Finding a study does not mean that someone will qualify to participate, and final eligibility is determined by the study team.
People considering participation in clinical research may also wish to discuss the study with their metabolic care team.
Find Current Information
These external sources are maintained independently of CanPKU+ and should be used for the latest study status, eligibility, locations and research-program updates.
HARMONY Study on ClinicalTrials.gov Pegtibatinase Program - Travere Therapeutics Travere Clinical Trials Health Canada Clinical Trial Search PortalLanguage: CanPKU+ provides plain-language research information to help our community understand emerging therapies. Clinical trial records and sponsor materials should be consulted for complete and current study information.
