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 <10 pmol/L). The copeptin level used for the two group stratification will be the blinded copeptin value obtained at baseline from the hospital phase prior to administration of tolvaptan.

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
North Carolina	5
Pennsylvania	5
Georgia	5
Virginia	4
Ohio	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, Market Analysis, and Projections

Introduction

Samsca, known generically as tolvaptan, is a selective vasopressin V2-receptor antagonist developed by Otsuka Pharmaceutical Co., Ltd. It is primarily used to treat hyponatremia (low sodium levels in the blood) and autosomal dominant polycystic kidney disease (ADPKD). Here, we will delve into the clinical trials, market analysis, and future projections for Samsca.

Clinical Efficacy and Trials

Hyponatremia Treatment

Samsca has demonstrated significant clinical efficacy in treating patients with hyponatremia, particularly those with the syndrome of inappropriate antidiuretic hormone secretion (SIADH) and other conditions such as heart failure and liver disease. Clinical trials have shown that Samsca effectively increases sodium levels in these patients, with close monitoring required to avoid rapid correction of hyponatremia, which can lead to osmotic demyelination syndrome[1][3].

ADPKD Trials

In clinical trials evaluating Samsca for the treatment of ADPKD, the drug showed promise but also raised concerns about liver safety. The FDA observed an increased risk of liver injury in these trials, leading to significant changes in the drug's labeling and usage guidelines[1].

Safety Concerns and Label Updates

Due to the risk of liver injury, the FDA limited the duration of Samsca treatment to 30 days and advised against its use in patients with underlying liver disease. The earliest case of severe liver injury was observed three months after initiation of tolvaptan, highlighting the need for careful patient selection and monitoring[1].

Market Approval and Launch

Global Launch

Samsca has been approved and launched in several regions, including the United States, Europe, and Japan. In the U.S., it was launched under the brand name JYNARQUE for the treatment of ADPKD in May 2018. In Europe, it is marketed as JINARC for the same indication[2][4].

Regulatory Environment

The FDA's restrictions on the use of Samsca due to liver injury risks have been a significant factor in its market dynamics. Despite these restrictions, the drug has maintained a strong market presence through careful patient management and monitoring[1].

Market Performance and Revenue Growth

Revenue Contribution

Samsca has been a substantial contributor to Otsuka's revenue growth. During the Third Medium-Term Management Plan (2019-2023), revenue from Samsca/JINARC/JYNARQUE saw significant increases, outperforming plan targets and contributing significantly to the company's consolidated performance. In fiscal 2019, revenue from these products rose by 65.1%[2][4].

Global Expansion

The drug has been launched in approximately 20 countries and has been prescribed to around 5,800 patients with ADPKD. Efforts to raise disease awareness and promote proper use guidelines have been instrumental in increasing prescriptions and driving revenue growth[2].

Market Dynamics and Competition

Therapeutic Franchises

Samsca operates within Otsuka's therapeutic franchises, which include treatments for psychiatric, neurological, and renal conditions. The company's strategy to diversify its revenue structure and strengthen its therapeutic franchises has helped Samsca maintain a competitive edge in the market[2].

Disease Awareness and Education

Otsuka's activities in promoting disease awareness, particularly for ADPKD, have been crucial in expanding the market reach of Samsca. These efforts have helped in increasing prescriptions and ensuring proper use of the drug[2][4].

Regulatory Considerations

FDA Restrictions

The FDA's limitations on the duration and usage of Samsca due to potential liver injury have been a critical regulatory consideration. The drug should not be used for longer than 30 days, and its use in patients with underlying liver disease is contraindicated[1].

European Regulations

In Europe, Samsca is subject to similar regulatory oversight, with guidelines emphasizing the need for careful patient monitoring and the avoidance of concomitant use with other treatments for hyponatremia that could increase the risk of rapid sodium correction[3].

Challenges and Opportunities

Business Environment

The healthcare industry is undergoing significant changes, including advances in basic science, increased focus on treatment costs, and the aging of populations. These changes present both challenges and opportunities for Samsca. Otsuka's ability to adapt to these changes and invest in new technologies and science will be crucial for the drug's continued success[2].

Expansion into New Areas

Otsuka is exploring new therapeutic areas and modalities, which could provide additional opportunities for Samsca. For example, the company is working on an ultrasound-based renal denervation system, which could address unmet needs in hypertension treatment and further expand Samsca's market[2].

Financial Trajectory

Revenue Projections

Under Otsuka's Fourth Medium-Term Management Plan (2024-2028), the company aims to further accelerate growth, targeting increased revenue and expanding into new therapeutic areas. Samsca is expected to continue playing a crucial role in this strategy, contributing to the company's growth objectives[2][4].

Market Growth Drivers

The growth of Samsca is driven by several factors, including the increasing awareness of ADPKD, the expansion into new markets, and the ongoing efforts to promote proper use guidelines. These factors are expected to continue driving revenue growth for the drug[2].

Key Takeaways

• Clinical Efficacy: Samsca is effective in treating hyponatremia and ADPKD, with significant clinical trial data supporting its use.
• Market Approval and Launch: Approved and launched in multiple regions, including the U.S. and Europe.
• Revenue Growth: Substantial revenue increases, contributing significantly to Otsuka's consolidated performance.
• Global Expansion: Launched in approximately 20 countries, with increasing prescriptions due to disease awareness efforts.
• Regulatory Considerations: FDA restrictions limit usage to 30 days due to potential liver injury risks.
• Market Dynamics: Strong therapeutic franchises and ongoing efforts in disease awareness and education support its market position.

FAQs

What is Samsca used for?

Samsca (tolvaptan) is used to treat clinically significant hyponatremia (low sodium levels in the blood) and autosomal dominant polycystic kidney disease (ADPKD)[1][2].

What are the FDA restrictions on Samsca?

The FDA has limited the duration of Samsca treatment to 30 days and advised against its use in patients with underlying liver disease due to the risk of liver injury[1].

How has Samsca performed in the market?

Samsca has seen substantial revenue growth, contributing significantly to Otsuka's consolidated performance. It has been launched in approximately 20 countries and prescribed to around 5,800 patients with ADPKD[2][4].

What are the potential side effects of Samsca?

Samsca can cause liver injury, and patients should be monitored for signs of liver disease. Other potential side effects include rapid correction of hyponatremia, which can lead to osmotic demyelination syndrome[1][3].

Is Samsca available in generic form?

As of the current date, Samsca is protected by patents, and there are no generic versions available. However, there are ongoing Paragraph IV challenges that may affect its patent status in the future[2].

Sources

1. FDA Drug Safety Communication: FDA limits duration and usage of Samsca (tolvaptan) due to possible liver injury[1].
2. DrugPatentWatch: When will the SAMSCA patents expire, and when will generic versions be available?[2].
3. European Medicines Agency: Samsca, INN-tolvaptan - European Medicines Agency[3].
4. Otsuka Pharmaceutical Co., Ltd.: Five-Year Growth Phase (2019–2023)[4].