Travere Therapeutics, Inc. provided regulatory updates for its sparsentan programs in IgA nephropathy (IgAN) and focal segmental glomerulosclerosis(FSGS), as well as its pegtibatinase program for classical homocystinuria (HCU). Sparsentan NDA for accelerated approval in IgAN on-track for PDUFA target action date of November 17, 2022The Company recently completed a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) currently under priority review for accelerated approval of sparsentan for thetreatment of IgAN. The FDA indicated that the NDA review process is proceeding as planned with no advisory committee meeting expected and that it remains on-track for the previously assigned Prescription Drug User Fee Act (PDUFA) target action date of November 17, 2022.Following FDA feedback on the ongoing pivotal Phase 3 DUPLEX Study, Company planning to pursue traditional approval of sparsentan for FSGS in 2023The Company also completed its planned Type A meeting with the FDA to discuss a potential submission for accelerated approval of sparsentan for FSGS.

In recently received final meeting minutes, the FDA acknowledged the high unmet need for approved therapies as well as the challenges in studying FSGS but indicated that the interim analysis from the ongoing pivotal Phase 3 DUPLEX Study conducted in 2021 together with the recent limited additional estimated glomerular filtration (eGFR) data-cut do not meet their threshold to support an application for accelerated approval in FSGS, which would be the first accelerated approval in FSGS. The FDA indicated that the DUPLEX Study as designed maintains the potential for full approval pending completion of the study and recommends that the Company pursue traditional approval based on two-year eGFR slope. The Company anticipates having topline data from the DUPLEX Study, including full two-year eGFR data, in the first half of 2023 and to be in position to submit an NDA for full approval in the second half of the year The Company and its partner Vifor Pharma are applying for conditional marketing authorisation of sparsentan for the treatment of IgAN in Europe.

The Company and its partner Vifor Pharma are submitting a Conditional Marketing Authorisation (CMA) application for sparsentan for the treatment of IgAN in Europe. A review decision on a potential approval is expected in the second half of 2023. Pending completion of the DUPLEX Study and data supportive of approval, a subsequent CMA variation of sparsentan for the treatment of FSGS is targeted for submission by the end of 2023.Pegtibatinase granted Breakthrough Therapy Designation for HCU.

The FDA recently granted Breakthrough Therapy Designation to pegtibatinase, the Company's novel investigation alenzyme replacement therapy being evaluated for the treatment of HCU. The Breakthrough Therapy Designation is supported by data from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase in patients with HCU, as well as data from the Company's ongoing natural history study. In the COMPOSE Study, treatment with 1.5mg/kg, twice weekly doses of pegtibatinase resulted in rapid and sustained reductions in total homocysteine (tHcy) through 12 weeks of treatment, including a 55.1% mean relative reduction in tHcy from baseline as well as maintenance of tHcy below a clinically meaningful threshold of 100 µmol.

As of the data cut-off, pegtibatinase has been generally well-tolerated. Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on a clinically significant endpoint(s). Drugs that receive Breakthrough Therapy Designation are eligible for Fast Track designation features, intensive guidance on an efficient drug development program, and organizational commitment from the FDA.