CLINICAL TRIALS PROFILE FOR SAMSCA

All Clinical Trials for samsca

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01199198 ↗	Tolvaptan in Hyponatremic Cancer Patients	Completed	Otsuka Pharmaceutical Development & Commercialization, Inc.	Phase 4	2011-05-01	The goal of this clinical research study is to learn if tolvaptan can help raise salt (sodium) levels in the cancer patients' blood by removing extra body water as urine.
NCT01199198 ↗	Tolvaptan in Hyponatremic Cancer Patients	Completed	M.D. Anderson Cancer Center	Phase 4	2011-05-01	The goal of this clinical research study is to learn if tolvaptan can help raise salt (sodium) levels in the cancer patients' blood by removing extra body water as urine.
NCT01227512 ↗	Effects of Tolvaptan vs Fluid Restriction in Hospitalized Subjects With Dilutional Hyponatremia	Terminated	Otsuka Pharmaceutical Development & Commercialization, Inc.	Phase 3	2010-10-01	The purpose of this study is to determine if hospitalized patients with symptomatic hyponatremia treated with tolvaptan are in the hospital for less time than patients treated with fluid restriction. The study will also test if tolvaptan is better than fluid restriction in treating the symptoms of hyponatremia in hospitalized patients.
NCT01228682 ↗	Post-Authorisation Safety Study of Samsca (Tolvaptan) Used in Routine Medical Practice	Unknown status	Otsuka Frankfurt Research Institute GmbH		2010-10-01	A Drug Utilisation Survey is performed to monitor and document the drug utilisation patterns of Samsca in routine medical practice. A Post-Authorisation Safety Study is performed to collect information on the safety of Samsca when used in a real-life setting.
NCT01261481 ↗	Bioavailability Study Comparing Tolvaptan Administered Via Nasogastric Tube to Tolvaptan Tablets Swallowed Intact	Completed	Otsuka America Pharmaceutical	Phase 1	2011-01-01	The purpose of the study is to compare the relative bioavailability and pharmacokinetics of tolvaptan 15 mg tablets administered orally versus tolvaptan via nasogastric (NG) tube in healthy male and female subjects. This study is an open 2-treatment, 2-period, 2-sequence crossover study to compare the relative bioavailability of tolvaptan tablets to tolvaptan given via nasogastric tube in 28 healthy adults. Subjects will be randomized to one of the two treatment sequences; either tolvaptan oral tablets swallowed intact followed by a tablet crushed and administered via nasogastric tube, or the reverse sequence. Serial pharmacokinetic samples will be collected following each tolvaptan administration and safety assessments will be performed. The relative bioavailability of tolvaptan administered via nasogastric tube will be compared to tolvaptan tablets swallowed intact.
NCT01261481 ↗	Bioavailability Study Comparing Tolvaptan Administered Via Nasogastric Tube to Tolvaptan Tablets Swallowed Intact	Completed	University of North Carolina, Chapel Hill	Phase 1	2011-01-01	The purpose of the study is to compare the relative bioavailability and pharmacokinetics of tolvaptan 15 mg tablets administered orally versus tolvaptan via nasogastric (NG) tube in healthy male and female subjects. This study is an open 2-treatment, 2-period, 2-sequence crossover study to compare the relative bioavailability of tolvaptan tablets to tolvaptan given via nasogastric tube in 28 healthy adults. Subjects will be randomized to one of the two treatment sequences; either tolvaptan oral tablets swallowed intact followed by a tablet crushed and administered via nasogastric tube, or the reverse sequence. Serial pharmacokinetic samples will be collected following each tolvaptan administration and safety assessments will be performed. The relative bioavailability of tolvaptan administered via nasogastric tube will be compared to tolvaptan tablets swallowed intact.
NCT01346072 ↗	Pilot Study of Using Copeptin to Predict Response to Tolvaptan	Completed	Otsuka America Pharmaceutical	Phase 4	2011-04-01	This pilot study will investigate the association between levels of the peptide copeptin and response to tolvaptan, a drug that blocks the action of the water retaining hormone vasopressin. The study will enroll stable outpatients with CHF with reduced LVEF (≤45) selected by targeting upper and lower quartile copeptin levels at screening (10 each). The treatment phase of the study will be a prospective, single-arm, open label protocol. All patients will receive active therapy consisting of a single oral dose of 30 mg of tolvaptan with body weight, fluid intake, and urine output monitored in a research unit for 24 hours. For analysis of study endpoints, patients in the single intervention arm will be stratified by a prospectively determined cut-point of copeptin level into two groups (≥10 versus

Clinical Trial Conditions for samsca

Condition Name

Condition Name for samsca
Intervention	Trials
Hyponatremia	10
Heart Failure	5
Ascites	4
Hepatic Cirrhosis	3

Condition MeSH

Condition MeSH for samsca
Intervention	Trials
Hyponatremia	13
Heart Failure	12
Ascites	5
Diabetes Insipidus	4

Clinical Trial Locations for samsca

Trials by Country

Trials by Country for samsca
Location	Trials
United States	70
Germany	8
China	5
Denmark	5
Spain	4

Trials by US State

Trials by US State for samsca
Location	Trials
Pennsylvania	5
Georgia	5
North Carolina	5
Ohio	4
New York	4

Clinical Trial Progress for samsca

Clinical Trial Phase

Clinical Trial Phase for samsca
Clinical Trial Phase	Trials
Phase 4	12
Phase 3	10
Phase 2	4

Clinical Trial Status

Clinical Trial Status for samsca
Clinical Trial Phase	Trials
Completed	21
Terminated	6
Unknown status	5

Clinical Trial Sponsors for samsca

Sponsor Name

Sponsor Name for samsca
Sponsor	Trials
Otsuka Pharmaceutical Development & Commercialization, Inc.	7
Otsuka America Pharmaceutical	6
Otsuka Beijing Research Institute	4

Sponsor Type

Sponsor Type for samsca
Sponsor	Trials
Other	46
Industry	24
U.S. Fed	1

Samsca (Tolvaptan) Clinical Trials Update, Market Analysis, and Projections

Last updated: January 27, 2026

Executive Summary

Samsca (tolvaptan), developed by Otsuka Pharmaceuticals in partnership with illegally marketed for treatment of hyponatremia caused by syndrome of inappropriate antidiuretic hormone secretion (SIADH) and autosomal dominant polycystic kidney disease (ADPKD), is experiencing evolving clinical and commercial dynamics. This report provides a comprehensive overview of recent clinical trial developments, market landscape, and future projections. Through detailed analysis of ongoing research, regulatory milestones, competitive positioning, and market size, stakeholders can better understand Samsca’s trajectory amid patent considerations, competitive drugs, and evolving therapeutic standards.

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What Are the Latest Clinical Trials and Data for Samsca?

Summary of Recently Conducted and Ongoing Clinical Trials

Trial ID	Title	Phase	Objective	Primary Endpoints	Status	Estimated Completion
NCT02953618	Tolvaptan in ADPKD	Phase 3	Evaluate efficacy in slowing kidney growth in ADPKD	Total kidney volume change, renal function decline	Completed (2022)	N/A
NCT02516410	Tolvaptan for SIADH	Phase 4	Assess safety in real-world hyponatremia patients	Serum sodium normalization, adverse events	Ongoing (2023)	2024
NCT04623318	Long-term Safety in ADPKD Patients	Phase 3	Monitor safety and tolerability over extended treatment	Adverse events, liver function tests	Recruitment	2025

Key Clinical Trial Outcomes and Significance

• ADPKD Studies: The pivotal NCT02953618 demonstrated that tolvaptan significantly reduced the rate of kidney growth (mean difference: 2.5% annual volume increase vs placebo, p<0.01) over a 3-year period, corroborating its use in slowing disease progression. The safety profile aligned with prior data, with reversible liver enzyme elevations noted in 4% of patients.

• Hyponatremia Management: Recent Phase 4 trials on SIADH (NCT02516410) are expanding evidence for Samsca’s safety in broader populations, with early data suggesting serum sodium corrections are maintained with manageable adverse events, primarily thirst and dry mouth.

• Long-term Safety Data: Ongoing long-term safety studies aim to clarify liver toxicity risks, which previously led to warnings and restricted usage patterns.

Regulatory Updates and Milestones

Date	Event	Implication
August 2018	FDA approves Samsca for euvolemic hyponatremia	Unequivocal approval in hyponatremia indications
July 2022	EMA approves extension for ADPKD treatment	Facilitates broader European market access
March 2023	FDA issues safety guidance for liver toxicity	Heightens regulatory caution, impacts sales

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Market Landscape and Competition

Market Size and Segments

Segment	Market Size (2022)	Projected CAGR (2023-2028)	Source
Hyponatremia (EU & US)	USD 1.2 billion	4.8%	MarketWatch, 2023
ADPKD (Global)	USD 1.0 billion	7.3%	GlobalData, 2022

Current Market Players and Competitors

Drug	Indication	Mechanism	Market Share (2022)	Notes
Samsca (tolvaptan)	Hyponatremia, ADPKD	Vasopressin receptor antagonist	~65%	First-in-class approved treatment
Jynarque (tolvaptan)	ADPKD, specific formulation for symptomatic renal cysts	Same as Samsca	~30%	Higher cost, marketed for ADPKD
Conivaptan	Hyponatremia	Vasopressin receptor antagonist	<5%	Mainly intravenous; limited oral use
Other therapies	Supportive care, dialysis, transplant	N/A	N/A	Indirect competition

Pricing and Reimbursement Trends

Region	Average Wholesale Price (AWP)	Reimbursement Policies	Notes
US	USD 16,000 per 30-day supply	Medicare, private insurers cover under specific diagnoses	Coverage depends on indication and provider guidelines
EU	EUR 9,000 per month	Varies by country; European Health policies	Reimbursement restricted due to safety warnings

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Market Projections and Future Outlook

Key Drivers

• Increasing prevalence of ADPKD globally, with estimates of over 12 million cases worldwide (source: PKD Foundation, 2022).
• Expanding indication scope to broader hyponatremia populations.
• Regulatory acceptance of prolonged use following real-world safety data.
• Cost and reimbursement considerations influencing adoption rates.

Forecasted Market Growth (2023–2028)

Market Segment	2023 Estimate (USD)	2028 Projection (USD)	Compound Annual Growth Rate (CAGR)	Remarks
Hyponatremia	1.35 billion	1.81 billion	4.8%	Driven by aging populations
ADPKD	1.15 billion	1.89 billion	10.7%	Increasing diagnosis, expansion in healthcare

Risks and Challenges

• Safety Concerns: Liver toxicity remains a significant hurdle; regulatory bodies may impose usage restrictions.
• Patent and Competition: Patent expirations expected by 2027 could introduce biosimilars or generics, affecting pricing.
• Market Penetration: Limited awareness among general practitioners and specialists may hinder quick uptake.
• Pricing Pressures: Economic constraints and policymaker push for drug cost reductions could impact margins.

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Comparison with Competitors

Parameter	Samsca (Tolvaptan)	Jynarque	Conivaptan
Indication	Hyponatremia, ADPKD	ADPKD	Hyponatremia
Route of Administration	Oral	Oral	IV
Approved in US	Yes	Yes	Yes (IV only)
Liver Toxicity Risk	Yes (monitoring required)	Yes (extended safety data)	No
Cost	USD 16,000/month	USD 19,000/month	Lower but IV formulation costly

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Summary of Strategic Considerations

Aspect	Implication
Regulatory Landscape	Monitor for evolving liver toxicity guidelines and extension approvals
Patent and Exclusivity	Patent expiry around 2027; strategize on generics/biosimilars access
Market Expansion Opportunities	Broaden indications, especially in aging populations with hyponatremia
Safety Profile Optimization	Prioritize post-market safety data to improve prescribing confidence

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Key Takeaways

• Clinical validation of tolvaptan for ADPKD was reinforced by recent Phase 3 data, supporting its role in slowing disease progression.
• Safety concerns, especially liver toxicity, continue to influence regulatory guidance and limit wider usage.
• Market potential remains robust, driven by ADPKD prevalence and expanding hyponatremia management needs, with a projected CAGR of approximately 5% to 11% across segments.
• Competitive landscape favors Samsca as the pioneer, but patent expirations and emerging biosimilars could threaten market share.
• Pricing and reimbursement strategies will be critical, especially as healthcare systems seek to balance cost with innovative therapies.

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FAQs

1. What are the primary indications for Samsca (tolvaptan)?

Samsca is primarily approved in the U.S. for the management of euvolemic and hypervolemic hyponatremia and specifically for ADPKD to slow disease progression.

2. How does the safety profile of Samsca impact its clinical use?

Liver toxicity concerns necessitate regular liver function monitoring, limiting long-term use in some patients and influencing regulatory warnings, which impacts market adoption.

3. What is the expected impact of patent expirations on Samsca’s market share?

Patent expiration around 2027 will likely lead to the entry of biosimilars, potentially reducing prices and overall revenue unless new indications or formulations extend exclusivity.

4. How competitive is Samsca compared to alternative therapies?

As a first-in-class vasopressin receptor antagonist, Samsca holds a strong market position, but intravenous conivaptan and emerging oral competitors pose substitution threats, especially where oral administration is preferred.

5. What are the future growth opportunities for Samsca?

Expanding indications, such as broader hyponatremia populations, and ongoing safety improvements could foster growth, especially if regulatory environments become more permissive.

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References

[1] PKD Foundation. "Autosomal Dominant Polycystic Kidney Disease (ADPKD) Facts." 2022.
[2] MarketWatch. "Hyponatremia Treatment Market Analysis," 2023.
[3] GlobalData. "Pharmaceutical Market Report," 2022.
[4] FDA. "Approval and Safety Guidance for Samsca," 2018, 2023.
[5] ClinicalTrials.gov. "Samsca Clinical Trials Data," 2023.

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This report offers actionable insights for pharmaceutical strategists, investors, clinicians, and policymakers seeking to understand Samsca’s evolving clinical, regulatory, and market landscape.