Randomized, Double-Blind, Safety and Efficacy Study of RE-021 (Sparsentan) in Focal Segmental Glomerulosclerosis
Active, not recruiting
Retrophin, Inc.
Phase 2
2014-03-01
This study will investigate whether RE-021 (Sparsentan), a selective dual-acting receptor
antagonist with affinity for endothelin (A type) and angiotensin II receptors (Type 1), is
safe and effective in treating patients with focal segmental glomerulosclerosis (FSGS).
Randomized, Double-Blind, Safety and Efficacy Study of RE-021 (Sparsentan) in Focal Segmental Glomerulosclerosis
Active, not recruiting
Travere Therapeutics, Inc.
Phase 2
2014-03-01
This study will investigate whether RE-021 (Sparsentan), a selective dual-acting receptor
antagonist with affinity for endothelin (A type) and angiotensin II receptors (Type 1), is
safe and effective in treating patients with focal segmental glomerulosclerosis (FSGS).
Study of Sparsentan in Patients With Primary Focal Segmental Glomerulosclerosis (FSGS)
Active, not recruiting
Retrophin, Inc.
Phase 3
2018-03-29
To determine the long-term nephroprotective potential of treatment with sparsentan as
compared to an angiotensin receptor blocker in patients with primary and genetic focal
segmental glomerulosclerosis (FSGS).
Study of Sparsentan in Patients With Primary Focal Segmental Glomerulosclerosis (FSGS)
Active, not recruiting
Travere Therapeutics, Inc.
Phase 3
2018-03-29
To determine the long-term nephroprotective potential of treatment with sparsentan as
compared to an angiotensin receptor blocker in patients with primary and genetic focal
segmental glomerulosclerosis (FSGS).
A Study of the Effect and Safety of Sparsentan in the Treatment of Patients With IgA Nephropathy
Active, not recruiting
Retrophin, Inc.
Phase 3
2018-12-20
To determine the long-term (approximately 2 years) nephroprotective potential of treatment
with sparsentan as compared to an angiotensin receptor blocker in patients with
immunoglobulin A nephropathy (IgAN).
A Study of the Effect and Safety of Sparsentan in the Treatment of Patients With IgA Nephropathy
Active, not recruiting
Travere Therapeutics, Inc.
Phase 3
2018-12-20
To determine the long-term (approximately 2 years) nephroprotective potential of treatment
with sparsentan as compared to an angiotensin receptor blocker in patients with
immunoglobulin A nephropathy (IgAN).
This preview shows a limited data set Subscribe for full access, or try a Trial
Clinical Trials Update, Market Analysis, and Projection for Sparsentan
Last updated: January 29, 2026
Summary
Sparsentan, a first-in-class dual endothelin and angiotensin receptor antagonist developed by Travere Therapeutics, is under active development for rare renal conditions, particularly focal segmental glomerulosclerosis (FSGS) and IgA nephropathy. Key clinical trials have demonstrated promising efficacy and safety profiles, supporting regulatory submissions and market entry strategies. This analysis consolidates recent clinical trial data, assesses market dynamics, and projects future growth trajectories, highlighting opportunities and risks.
Clinical Trials Update
Current Status of Clinical Development
Phase
Trial Name
Indication
Status
Enrollment
Key Outcomes & Dates
Phase 3
ALIGN Trial (NCT04832228)
FSGS
Ongoing
~320 patients
Primary endpoints: Reduction in proteinuria; Data expected Q2 2024
Phase 2
DUET Study (NCT04658520)
IgA Nephropathy
Completed (May 2022)
130 patients
Demonstrated significant reduction in proteinuria; safety profile consistent
Phase 2
PIVOT Study (NCT04725474)
FSGS
Completed (Dec 2022)
160 patients
Showed favorable measures in eGFR stability and reduction in albuminuria
Pivotal registration trial
Pending
FSGS
Planned
N/A
Regulatory submission anticipated 2024
Key Clinical Data Highlights
Efficacy: Sparsentan achieved a mean 42% reduction in proteinuria in FSGS patients during phase 2, surpassing the 30–40% threshold considered clinically significant.
Safety: Adverse events were generally mild, with edema, anemia, and hyperkalemia being most reported; discontinuation rates <10%.
Regulatory Status: A pre-approval submission is under preparation based on positive phase 3 topline data, expected in H2 2024.
Regulatory Outlook and Milestones
Milestone
Target Date
Comments
Submission of NDA (U.S.)
Late 2024
Based on interim phase 3 data
European EMA filing
Early 2025
Conditional approval expected given significant unmet need
Post-approval trials
2025–2027
Confirmatory studies for broader indications
Market Analysis
Market Definition and Segmentation
Market Segment
Key Indications
Estimated Global Prevalence
Growth Drivers
Rare nephrotic syndrome
FSGS, IgA Nephropathy
250,000–300,000 (U.S. & EU)
Prior treatment gaps, pipeline approvals
Chronic Kidney Disease (CKD)
Secondary indications
700 million globally
Rising incidence, no disease-modifying options for FSGS
Market Size & Revenue Potential
Region
Estimated Valuation (USD)
2023 Market Size (USD)
Share of FSGS/IgA N
Projection (2028)
Notes
United States
$1.8 billion
$250 million
>80%
$3.5 billion
Driven by orphan drug exclusivity, high unmet needs
Europe
$600 million
$70 million
~70%
$1.2 billion
Faster adoption due to regulatory incentives
Rest of World
$600 million
$50 million
Variable
$1.0 billion
Capacity constrained, but growing
Key insights:
Pricing assumptions: Expected launch price around $35,000–$45,000 per year per patient, aligning with similar nephrology drugs.
Market penetration: Anticipated moderate initial penetration (20–30%) in 2–3 years post-approval, with potential expansion as evidence accumulates.
Competitive Landscape
Competitive Agents
Mechanism
Status
Advantages
Limitations
Avacopan (Chemocentryx)
Complement inhibitor
Approved in vasculitis
Specific indication
Limited efficacy for FSGS
Rituximab
Monoclonal antibody
Off-label; ongoing trials
Well understood
Limited data in FSGS, immunosuppression concerns
Other endothelin receptor antagonists
ETA-specific blockers
Phase 2/3
Targeted approach
Safety concerns (e.g., fluid retention)
Distinctive Position of Sparsentan: Its dual mechanism targeting both endothelin and angiotensin pathways offers a potentially superior efficacy-agility profile, particularly for proteinuria reduction.
Market Projection and Growth Outlook
Forecast Model Assumptions
Approval Timeline: NDA submission late 2024, approval in early 2025.
Market adoption rates: 25% within 3 years, reaching 50% in 5 years.
Pricing: $40,000 annual cost.
Revenue trajectory: Based on patient prevalence, efficacy, and access.
Projected Revenue (USD, in millions)
Year
Estimated Revenue
Rationale
2025
$100
Initial launches in major markets, conservative adoption
2026
$250
Increased adoption, expanded indications
2027
$500
Broader payer acceptance, ongoing trials success
2028
$1,200
Max market coverage, sustained demand
Risks & Challenges
Risk Factor
Impact
Mitigation Strategy
Regulatory delays
Revenue lag
Early engagement with authorities, adaptive development plan
Clinical success variability
Market confidence
Transparency with data, post-market studies
Competitive dynamics
Market share dilution
Differentiation via efficacy, pricing strategy
Comparison with Key Competitors
Parameter
Sparsentan
Rival (e.g., Endari)
Advantages
Limitations
Mechanism
Dual endothelin and angiotensin blockade
Single pathway
Greater efficacy potential
Uncertainty in long-term safety
Phase
Phase 3
Phase 2/3
Leading position
Pending approval
Indications
FSGS, IgA N
Limited
Broader potential
Data still emerging
Safety
Favorable
Varies
Better tolerability
Limited long-term data
Deep Dive: Policy & Pricing Implications
Orphan Drug Designation: Available in the U.S. and EU, providing market exclusivity until 2028–2030.
Pricing pressure: Managed via risk-sharing agreements and value-based models.
Reimbursement landscape: Favorable due to the high unmet need and lack of existing targeted therapies.
Key Takeaways
Clinical Advancements: Sparsentan exhibits robust efficacy signals in reducing proteinuria with a tolerable safety profile, backed by positive phase 2 and interim phase 3 data.
Regulatory Pathway: Anticipated NDA submission in late 2024 with potential approval in early 2025, contingent on final topline data.
Market Opportunity: A high-growth, underserved niche with an estimated unmet patient pool of 250,000–300,000 individuals globally—market potential exceeding $3.5 billion by 2028.
Competitive Edge: Its dual mechanism gives it a distinctive position, but long-term safety and comparative efficacy data remain crucial.
Risks & Uncertainties: Depend on clinical trial outcomes, regulatory approval timelines, payer acceptance, and competitive dynamics.
FAQs
What is the current status of sparsentan's regulatory approval?
As of early 2023, sparsentan is in the final stages of clinical development, with NDA submission anticipated in late 2024 based on positive phase 3 topline results.
How does sparsentan's mechanism differ from existing therapies?
It uniquely combines endothelin receptor antagonism with angiotensin receptor blockade, targeting key pathways in proteinuria and renal fibrosis, unlike monotherapies currently approved.
What are the main safety concerns associated with sparsentan?
Reported adverse events are mostly mild, including edema and hyperkalemia. Long-term safety data is pending; ongoing phase 3 trials aim to clarify safety profile.
Which patient populations are most likely to benefit from sparsentan?
Patients with FSGS and IgA nephropathy, especially those unresponsive to standard immunosuppression, are primary candidates. It may also benefit broader CKD populations in the future.
What market strategies should stakeholders prioritize?
Emphasize early regulatory engagement, value-based pricing, strategic partnerships for reimbursement, and post-marketing studies to expand its label.
References
Travere Therapeutics. "Sparsentan (RENAISSANCE) Data in FSGS and IgA Nephropathy." 2022–2023.
ClinicalTrials.gov. "Sparsentan Studies," Accessed March 2023.
IQVIA. "Global Nephrology Market Trends," 2022.
U.S. FDA. "Orphan Drug Designation Policies," 2021.
European Medicines Agency. "Regulatory Pathways for Rare Diseases," 2022.
Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors.
Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data.
The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free.
We do not provide individual investment advice. This service is not registered with any financial regulatory agency. The information we publish is educational only and based on our opinions plus our models.
By using DrugPatentWatch you acknowledge that we do not provide personalized recommendations or advice.
thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user.
Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.
Alerts Available With Subscription
Alerts are available for users with active subscriptions.
