Benzinga | May 25, 2021 04:05PM EDT

Travere Therapeutics Provides Regulatory Update On Sparsentan Development Program For Focal Segmental Glomerulosclerosis

Travere Therapeutics, Inc. (NASDAQ:TVTX) today provided a regulatory update for its sparsentan program in focal segmental glomerulosclerosis (FSGS). Following achievement of the interim proteinuria endpoint in the ongoing Phase 3 DUPLEX Study of sparsentan in FSGS, the Company conducted pre-New Drug Application (NDA) interactions with the U.S. Food and Drug Administration in pursuit of an accelerated approval submission in the U.S., and Marketing Authorization Application (MAA) pre-submission interactions with the European Medicines Agency (EMA) for conditional marketing authorization and accelerated assessment consideration in Europe.



In recently received final pre-NDA meeting minutes, the FDA acknowledged the high unmet need for approved therapies for the treatment of FSGS but indicated the available data from the interim assessment of the DUPLEX Study would not be adequate to support an accelerated approval at this time. Based upon this feedback, the Company no longer expects to submit for accelerated approval for FSGS in the U.S. during the second half of 2021. The FDA has indicated that it may be possible to submit an application for accelerated approval after additional data accrue in the study. Subject to further discussion with the FDA, the Company believes that it may be possible to provide sufficient additional estimated glomerular filtration (eGFR) data from the DUPLEX Study in the first half of 2022. The FDA has encouraged the Company to request a follow-up meeting to further explore this option in greater detail, and a Type A meeting is expected to occur in the third quarter of 2021.

"We remain very confident in the profile of sparsentan and believe it supports the ability to ultimately become a new treatment standard for FSGS, but we are disappointed that we will not be able to submit for accelerated approval this year and deliver it to patients on our original targeted timeline," said Eric Dube, Ph.D., chief executive officer of Travere Therapeutics. "Based upon our ongoing dialogue with FDA, we believe the eGFR data need to further mature to support our New Drug Application for accelerated approval in this indication. We remain optimistic that we still have a potential opportunity to pursue a path to an accelerated approval submission in the U.S. next year. We look forward to continuing our collaborative discussions with the FDA and providing a further update in the third quarter of this year."

The Company also conducted initial MAA pre-submission interactions with the EMA. In that meeting, the Company received administrative support for proceeding as planned with a conditional marketing authorization submission in the second half of 2021. The agency also provided direction on administrative and regulatory topics, as well as guidance for upcoming discussions with assigned rapporteurs and co-rapporteurs. As a result of these interactions, the Company will continue its preparations for an MAA submission of sparsentan in FSGS and seek confirmation of its plans with the assigned rapporteurs and co-rapporteurs in an upcoming meeting.