Executives from Travere Therapeutics (NASDAQ:TVTX) said the company is focused on advancing treatments in three rare diseases—IgA nephropathy (IgAN), focal segmental glomerulosclerosis (FSGS), and homocystinuria (HCU)—as management discussed regulatory timelines, commercial momentum, and capital resources at Guggenheim’s Biotech Summit.
Company overview: three rare disease priorities
President and CEO Eric Dube said Travere is “focused exclusively on rare disease,” with efforts concentrated in IgAN, FSGS, and HCU. He described 2025 as an active period across programs, highlighting continued commercialization of FILSPARI in IgAN, an FDA review for an FSGS supplemental NDA (sNDA) for FILSPARI, and plans to restart the Phase 3 HARMONY study of pegtibatinase in HCU after resolving manufacturing scale-up challenges.
FSGS: FDA review, April PDUFA, and expectations for uptake
The company previously disclosed an extension of the PDUFA date to April 13 after the FDA requested additional information. Dube said the information requests arrived in December, at a point when the company had been expecting draft labeling, and described the questions as “quite straightforward,” though he said it was unclear why they came late in the review. He added that Travere responded quickly and believes it has answered the agency’s questions, and is now waiting for draft labeling and a potential approval decision on April 13.
Asked about FDA staffing changes and whether turnover could have contributed to the late questions, Dube said the company saw “a number of new reviewers” during the December exchanges, but noted that because the filing is an sNDA, Travere does not have formal mid-cycle or late-cycle meetings, making it difficult to assess the degree of personnel change and its impact. He said Travere continues to have strong engagement with the FDA and noted that the director of the cardiorenal division who has been involved in the program for over 10 years “is still there.”
From a communications standpoint, Dube said the company expects to enter a quiet period about a month before the PDUFA date—sometime in early March after upcoming earnings—and remain quiet until the PDUFA date or other meaningful written correspondence from the FDA.
On launch readiness, Dube said Travere has expanded its field team and intends to be prepared to move quickly if approved, citing what he described as urgency within the FSGS community. He said the company expects “rapid uptake,” supported by physicians’ familiarity with FILSPARI in IgAN. He also said there is roughly a 90% overlap between physicians treating IgAN and FSGS, with a key difference being pediatric nephrologists; he said Travere would expect an FSGS approval to include pediatric patients and that the company’s expansion includes reaching pediatric specialists.
On competition, Dube said he would not be surprised if others follow the regulatory pathway the company helped establish. He added that he does not expect mechanisms in development to be curative, and suggested patients may need multiple treatment options and likely combination therapy. He said Travere does not currently see anything in development with the same mechanism as sparsentan and estimated it would be “a number of years” before other products become commercially available.
IgA nephropathy: commercial momentum, combination use, and REMS changes
Discussing IgAN, Dube said Travere is closing its “third year of launch” of FILSPARI and is working to position it as “foundational care,” replacing what he described as the traditional role of RAS inhibitors and ARBs. He said the company ended the prior year with its strongest commercial quarter to date, attributing some of the performance to the expanded field team that came on in the fall.
With new therapies entering the IgAN landscape, Dube said more options are welcomed by the community and may support a future where kidney failure can be avoided through earlier and more aggressive treatment and combination therapy. He said Travere is seeing FILSPARI used in earlier, less severe patients and noted that treatment guidelines call for earlier intervention when patients are not at goal, which he described as essentially complete remission. He also said the company is seeing early, anecdotal evidence of FILSPARI being used in combination with newer therapies, consistent with KDIGO guideline alignment, and added that Travere has not seen evidence that new entrants are dampening FILSPARI’s growth, instead suggesting that increased awareness is expanding the market.
On payer behavior, Dube said it remains early but that Travere is seeing payers cover these therapies and has anecdotal examples of payers allowing combination therapy on top of FILSPARI. He emphasized that payer behavior can vary. He also argued Travere has a unique positioning versus immune-targeted therapies and highlighted health economic evidence, including that Travere is the only company he said has run a head-to-head study against an active comparator, positioning FILSPARI as a superior choice versus ACE inhibitors or ARBs.
Regarding patient persistence, Dube said refill rates have been high for a chronic therapy, attributing this to once-daily dosing and patient-perceived improvements in lab values.
On the FILSPARI REMS program, Dube said a modification in fall 2024 that reduced testing frequency from monthly to quarterly was a meaningful contributor to commercial performance, aligning with typical nephrology visit cadence. He said the REMS still plays a role in maintaining awareness of the product profile and ensuring periodic lab monitoring, and that the company will continue building its safety data with the eventual goal of discussing REMS removal. He noted that pregnancy monitoring was removed as part of the prior fall change, and added that Travere has wanted to maintain focus on the FSGS approval process before engaging the FDA on further REMS modifications.
HCU: pegtibatinase and Phase 3 restart plans
Dube described homocystinuria as a genetic metabolic disease caused by defects in the CBS enzyme, leading to elevated homocysteine and methionine levels that can become toxic over time. He outlined potential consequences including cognitive and psychiatric effects, eye issues such as lens dislocation, and thrombotic risk, stating that about 25% of affected children will have an ischemic event before their teenage years and about half before age 30.
He said current management centers on dietary protein restriction and replacement with medical protein, along with vitamin B6 for patients who retain some enzyme activity, adding that about half of patients are responsive to B6. Dube said there are about 10,000 diagnosed patients in the U.S., with approximately 3,500 actively under care and not at goal, but also suggested that number may be underestimated because he believes about half of patients are not diagnosed despite newborn screening.
Travere said manufacturing scale-up challenges from more than a year ago have been resolved, enabling the company to restart the Phase 3 HARMONY study this quarter. Dube said the company expects enrollment to be quick, while noting a key trial consideration is managing variability driven by diet. He said the study includes an extensive screening period of more than eight weeks to stabilize diet before dosing. Travere said it typically provides guidance on the timeline for top-line data after first patient dosing, indicating that update would come later.
On competition in HCU, Dube said the company does not see anything in clinical development, though Travere still intends to move quickly.
Patent outlook, business development, and cash position
CFO Chris Cline addressed questions on FILSPARI’s patent estate, stating that the company’s base case is exclusivity through 2033. He said this expectation is grounded in an Orange Book-listed patent with patent term extension and orphan drug exclusivity for each indication, and added that additional work underway with PTO review could extend protection beyond 2033.
Cline also said Travere is interested in business development after the FSGS process, contingent on internal capacity, with a focus on assets that have synergies with the company’s mid-to-late-stage development and commercial capabilities.
On liquidity, Cline said Travere ended the year with $323 million in cash and does not see a near-term need for capital. He said the company believes it can fund its priorities of maintaining IgAN momentum, pursuing approval and launch execution in FSGS, and advancing pegtibatinase toward potential commercialization.
About Travere Therapeutics (NASDAQ:TVTX)
Travere Therapeutics, Inc (NASDAQ: TVTX) is a biopharmaceutical company headquartered in San Diego, California, dedicated to the development and commercialization of therapies for rare kidney and genetic disorders. The company’s mission is to address unmet needs in conditions with limited treatment options by focusing on diseases that affect small patient populations. Travere combines research, development and commercial capabilities to bring innovative medicines to market.
The company’s lead product is sparsentan, a dual endothelin angiotensin receptor antagonist that has received accelerated approval from the U.S.
