Travere Therapeutics: Enzyme Replacement Therapy


Some people may know of this drug as OT-58, but its proper name is TVT-058, Pegtibatinase.

In November 2020, Retrophin acquired Orphan Technologies, thus also acquiring OT-58 and their Natural History Study. Shortly after, Retrophin rebranded as Travere.

Travere Therapeutics is in the process of developing an enzyme replacement therapy for patients with CBS Deficiency Homocystinuria.

For the most up-to-date news on their enzyme replacement, please visit their website: Click here

May 31, 2023

Travere Therapeutics Announces Positive Topline Results from Cohort 6 in the Phase 1/2 COMPOSE Study of Pegtibatinase in Classical Homocystinuria

  • Data from the four patients treated with the highest dose of pegtibatinase showed a clinically meaningful 67.1% mean relative reduction in total homocysteine from baseline
  • Pegtibatinase has been generally well-tolerated to date
  • The Company is engaging with regulators and expects to initiate a pivotal Phase 3 Study by year-end 2023 

October 27, 2022

Travere Therapeutics Reports Third Quarter 2022 Financial Results and
Corporate Updates

Travere Therapeutics, Inc.  today reported its third quarter 2022 financial results and provided a corporate update.

A Breakthrough Therapy Designation was granted to the pegtibatinase development program for classical homocystinuria (HCU).

August 25, 2022

Travere Therapeutics to Present Abstracts at the Society for the Study of Inborn Errors of Metabolism Annual Symposium

  • Population-based Incidence Estimates of Classical Homocystinuria Using the Genome Aggregation Database (gnomAD)

  • A Longitudinal Study of Cognitive Function in Classical Homocystinuria Demonstrates Distinct Deficits in Inhibitory Control

  • Insights from the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)

August 3, 2022

Travere Therapeutics Provides Regulatory Updates on its Development Programs

  • Pegtibatinase granted Breakthrough Therapy Designation by FDA