BioMarin for a diverse portfolio of rare disease therapies.

A specialized biotech like Vertex, but for a wider array of rare conditions.

```html

• Chenodal: An oral medication used to treat radiolucent stones in gallbladders.
• Cholbam: A cholic acid capsule prescribed for pediatric and adult patients with bile acid synthesis disorders and as an adjunctive treatment for peroxisomal disorders.
• Thiola / Thiola EC: Tiopronin tablets used for the treatment of homozygous cystinuria.
• Sparsentan: A product candidate in Phase III clinical trials for the treatment of focal segmental glomerulosclerosis and immunoglobulin A nephropathy.
• TVT-058: A novel investigational human enzyme replacement candidate in Phase I/II clinical trials for classical homocystinuria.

```

Travere Therapeutics, Inc. (TVTX) is a biopharmaceutical company that develops and commercializes therapies for rare diseases. As such, its primary customers are not individual patients directly, but rather various entities within the healthcare supply chain that facilitate the distribution and dispensing of its prescription drugs.

The major categories of customers for Travere Therapeutics (TVTX) include:

1. Pharmaceutical Wholesalers and Distributors: These companies purchase drugs in bulk from manufacturers like Travere Therapeutics and distribute them to pharmacies, hospitals, and other healthcare providers. Examples of such public companies include AmerisourceBergen (NYSE: ABC), Cardinal Health (NYSE: CAH), and McKesson Corporation (NYSE: MCK), although specific contractual relationships are not disclosed in the company description.
2. Specialty Pharmacies: Given Travere's focus on rare diseases, its products often require specialized handling, storage, and patient support, making specialty pharmacies a crucial distribution channel.
3. Hospitals and Clinics: Healthcare institutions may directly purchase Travere's therapies for administration to patients within their facilities or for dispensing through their own outpatient pharmacies.

null

Eric Dube, Ph.D. President & Chief Executive Officer Dr. Dube has served as President and Chief Executive Officer of Travere Therapeutics since January 2019. Prior to joining Travere, he was president and head of North America at ViiV Healthcare. He also spent over 18 years in various leadership roles at GlaxoSmithKline plc (GSK), including leading the US Oncology and Global Respiratory businesses. Dr. Dube is a co-founder of OUTBio San Diego, a group for LGBTQ biotech professionals. Chris Cline, CFA Chief Financial Officer Mr. Cline serves as Chief Financial Officer and a member of the executive team at Travere Therapeutics, having assumed the position in August 2022. He is responsible for corporate and strategic finance and investor relations. Mr. Cline joined Travere in 2014, previously serving as Senior Vice President, Investor Relations and Corporate Communications. Before Travere, he was part of the global investor relations group at Elan Corporation, plc (which was acquired by Perrigo Company), and the financial planning and analysis group at Phase Forward, a healthcare technology company focused on growth through mergers and acquisitions. Jula Inrig, M.D. Chief Medical Officer Dr. Inrig joined Travere Therapeutics in January 2022 as Chief Medical Officer. She oversees the company's medical functions, clinical development, clinical operations, and drug safety/pharmacovigilance. With over 15 years of expertise in drug development and global regulatory engagement, Dr. Inrig previously served as Global Head of the Renal Center of Excellence at IQVIA, where she was instrumental in clinical trials leading to FDA and EMA approvals. Peter Heerma Chief Commercial Officer Mr. Heerma has served as Chief Commercial Officer at Travere Therapeutics since October 2019. He is responsible for leading the company's commercial organization and commercialization strategy for approved products and pipeline programs. He brings over 20 years of global experience in launching therapies and managing product portfolios across various therapeutic areas at top biopharmaceutical organizations. Prior to Travere, Mr. Heerma was Global Product General Manager for oncology and cardiovascular products at Amgen, and held commercial leadership roles at AbbVie and Abbott Laboratories. Casey Logan Chief Business Officer Mr. Logan is Chief Business Officer at Travere Therapeutics, a role he has held since 2018. He leads corporate development, strategic planning, program management, and information technology. Before joining Travere, Mr. Logan was chief business officer at Tracon Pharmaceuticals, where he led strategic, business, and corporate development efforts, including the company's IPO in January 2015. His prior experience includes senior corporate development roles at Bird Rock Bio and Anadys Pharmaceuticals, Inc. (acquired by Roche), and a position in the corporate business development group at Eli Lilly and Company.

The key risks for Travere Therapeutics (TVTX) primarily revolve around the success and market acceptance of its lead drug, FILSPARI (sparsentan), and the competitive landscape for its target indications.

1. Regulatory Approval and Commercialization of FILSPARI for FSGS: The approval and successful commercialization of FILSPARI for Focal Segmental Glomerulosclerosis (FSGS) is a critical near-term catalyst and a significant binary risk for Travere Therapeutics. While FILSPARI is fully approved for IgA nephropathy (IgAN), the FSGS indication represents a substantial expansion of the drug's addressable market and is crucial for the company's future revenue growth. The U.S. Food and Drug Administration (FDA) recently extended its review timeline for the supplemental New Drug Application (sNDA) for FILSPARI in FSGS to April 13, 2026, due to requests for additional information regarding clinical benefit, underscoring the ongoing regulatory hurdles. The company's future prospects are highly reliant on the successful commercialization of FILSPARI and the approval of sparsentan for FSGS.
2. Intensifying Competition in the IgA Nephropathy (IgAN) Market: The competitive landscape for IgAN treatments is intensifying, posing a significant challenge to FILSPARI's market share. Competitors, such as Vertex Pharmaceuticals, have reported strong Phase 3 interim data for rival IgAN drugs (e.g., povetacicept), which directly compete with FILSPARI and have already exerted pressure on Travere's stock. The emergence of new therapies, including SGLT2 inhibitors and other pathway agents, could further fragment the market and introduce headwinds for FILSPARI over time. As a smaller company, Travere may face challenges in marketing and distribution against larger pharmaceutical firms with more extensive resources.
3. Hepatotoxicity and Other Safety Considerations for FILSPARI: Despite an FDA-approved modification to its Risk Evaluation and Mitigation Strategy (REMS) that reduces the frequency of liver function monitoring, FILSPARI carries a risk of hepatotoxicity. Clinical studies have observed elevations in aminotransferases (ALT or AST) in a percentage of FILSPARI-treated patients, and some other Endothelin Receptor Antagonists (ERAs) have been associated with liver failure. Due to this risk, FILSPARI remains available only through a restricted REMS program. Other safety concerns include the potential for acute kidney injury and hyperkalemia, which require periodic monitoring. While these risks are being managed, they remain important considerations that could influence prescriber and patient acceptance.

null

Travere Therapeutics (TVTX) focuses on developing and commercializing therapies for rare diseases. The addressable markets for its main products and pipeline candidates are detailed below:

Sparsentan

Sparsentan is in Phase III clinical trials for the treatment of focal segmental glomerulosclerosis (FSGS) and immunoglobulin A nephropathy (IgAN). [cite: Background]

• Focal Segmental Glomerulosclerosis (FSGS): The global FSGS market size was valued at approximately USD 14.75 billion in 2024 and is projected to reach USD 27.71 billion by 2032, growing at a compound annual growth rate (CAGR) of 8.20% during the forecast period. Another estimate places the global FSGS market at USD 15.2 billion in 2026. The FSGS treatment market is expected to grow from USD 14.95 billion in 2025 to USD 15.9 billion in 2026, with a forecast to reach USD 21.63 billion by 2031 at a CAGR of 6.34%. North America held the largest market share in 2020, and the U.S. market alone accounted for 77% of North America's revenue in 2024. The market for Sparsentan tablets specifically is projected to expand from USD 2.4 billion in 2024 to USD 7.1 billion by 2035, with a CAGR between 7.7% and 11%, primarily driven by the increasing prevalence of IgA Nephropathy and advancements in dual-acting receptor blockers. Key growth regions for Sparsentan include the U.S., Canada, UK, Australia, and Germany.

• Immunoglobulin A Nephropathy (IgAN): The IgA Nephropathy (IgAN) market across the seven major markets (7MM - United States, EU4, UK, and Japan) was valued at approximately USD 730 million in 2024 and is anticipated to grow with a significant CAGR of 30.5% to reach USD 9,655 million by 2034. The global IgA Nephropathy market is projected to grow from USD 3.8 billion in 2024 to USD 5.8 billion by 2030, at a CAGR of 6.5%. In 2024, the U.S. IgA Nephropathy market was valued at approximately USD 455 million and is expected to increase with the introduction of new therapies. It is estimated to reach USD 2.12 billion by 2030 in the U.S.

Thiola and Thiola EC

Thiola and Thiola EC are tiopronin tablets indicated for the treatment of homozygous cystinuria. [cite: Background]

• Homozygous Cystinuria: The global cystinuria treatment market was valued at USD 160.00 million in 2024 and is projected to experience a CAGR of 4.70% to reach USD 210.77 million by 2030. Another estimate indicates the cystinuria market size was USD 907.19 million in 2024 and is anticipated to grow at a CAGR of 14.80% to achieve a value of USD 3.60 billion by 2034. The tiopronin category holds the largest market share in cystinuria treatment. The global cystinuria patient base is estimated to be around 10,000–15,000 in the EU and U.S. The United States is expected to lead the global market for cystinuria during the forecast period due to the increased prevalence of the condition and ongoing research and development.

TVT-058

TVT-058 is a novel investigational human enzyme replacement candidate in Phase I/II clinical trials for the treatment of classical homocystinuria. [cite: Background]

• Classical Homocystinuria (HCU): The global homocystinuria market is anticipated to expand from USD 66.3 million in 2024 to USD 95.3 million by 2034, growing at a CAGR of approximately 4.3%. Another source projects the global homocystinuria market size to reach US$ 94.50 million by 2034 from US$ 66.25 million in 2025, with a CAGR of 4.03% during 2026–2034. North America leads this market due to advanced healthcare infrastructure and increased awareness, with Europe also experiencing substantial growth. TVT-058 (Pegtibatinase) revenue is expected to reach an annual total of USD 81 million by 2038 in the U.S. Classical homocystinuria affects approximately 1 in 200,000 to 335,000 births globally.

Travere Therapeutics (TVTX) is poised for future revenue growth over the next 2-3 years, driven primarily by the expanding commercialization of its key product, FILSPARI, its potential label expansion, and the advancement of its pipeline. The expected drivers of future revenue growth include: * **Continued Growth of FILSPARI in IgA Nephropathy (IgAN)**: FILSPARI (sparsentan) has demonstrated robust commercial momentum since its launch for IgA nephropathy. In the fourth quarter of 2025, U.S. net product sales of FILSPARI reached $103.3 million, marking a 108% increase compared to the prior-year period. Total U.S. net product sales for FILSPARI for the full year 2025 were $322.0 million, representing a 144% growth over 2024. The company reported a record 908 new patient start forms (PSFs) in Q4 2025, indicating strong and sustained demand from both new and existing prescribers. * **Potential Launch of FILSPARI for Focal Segmental Glomerulosclerosis (FSGS)**: Travere Therapeutics is actively preparing for the potential commercial launch of FILSPARI for the treatment of focal segmental glomerulosclerosis (FSGS), a rare and serious kidney disorder with no currently approved therapies. The Prescription Drug User Fee Act (PDUFA) target action date for FILSPARI in FSGS is April 13, 2026. A potential approval in FSGS is anticipated to significantly expand FILSPARI's addressable market and serve as a substantial revenue driver. * **International Expansion of FILSPARI**: Travere is expanding FILSPARI's reach into international markets through strategic partnerships. CSL Vifor, a collaborator, has already launched FILSPARI in several European countries, including Germany, Austria, Switzerland, Luxembourg, and the UK, which entitles Travere to potential market access and sales-based milestone payments and tiered royalties. Additionally, Chugai Pharmaceutical, another partner, is expected to submit a New Drug Application for sparsentan in Japan in 2026, further extending the drug's global market presence. * **Advancement of Pegtibatinase (TVT-058) for Classical Homocystinuria (HCU)**: Enrollment activities have resumed for the pivotal Phase 3 HARMONY Study of pegtibatinase (TVT-058) in classical homocystinuria (HCU) in the first quarter of 2026, following optimization of its manufacturing process. This product candidate represents a significant opportunity as it could become the first potentially disease-modifying therapy for patients living with classical HCU, contributing to future revenue streams if successfully developed and approved.

Share Repurchases

• Travere Therapeutics did not repurchase any of its equity securities during the fiscal year ending December 31, 2025.

Share Issuance

• The company conducted public offerings of common stock in 2023 and 2024 to raise substantial funds.
• A common stock offering in November 2024 generated $134.7 million in net proceeds.
• The number of shares outstanding increased from 61.61 million at the end of 2021 to 89.23 million by the end of 2025.

Inbound Investments

• On October 6, 2025, PERCEPTIVE ADVISORS LLC significantly increased its stake in Travere Therapeutics by acquiring 4,061,363 shares at a price of $26.13.
• Travere Therapeutics raised a total of $275 million from a Post IPO funding round on March 8, 2022.

Outbound Investments

• Travere Therapeutics had not made any investments or acquisitions as of February 21, 2026.

Capital Expenditures

• In the third quarter of 2025, Travere Therapeutics invested $140,000 in capital expenditures, primarily for funding long-term assets and infrastructure.