CLINICAL TRIALS PROFILE FOR DEMSER

All Clinical Trials for DEMSER

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00028106 ↗	131MIBG to Treat Malignant Pheochromocytoma	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	2001-12-05	This study will evaluate the effectiveness of 131MIBG in treating malignant pheochromocytoma and whether sensitization medications improve the response to treatment. Pheochromocytoma is a rare type of tumor that usually occurs in the adrenal glands. The tumor cells release chemicals like adrenaline that can cause large increases in blood pressure and pulse rate, with serious health consequences. Tumor in the adrenal glands usually can be removed surgically, but if the pheochromocytoma is malignant-i.e., has spread to many sites in the body-or is located in places where surgery is difficult or impossible, no satisfactory treatment is available. 131MIBG is a combination of an adrenaline-like chemical and a radioactive form of iodine. The 131MIBG attaches to the tumor cells and the high concentration of radioactive iodine kills them. Previous studies using 131MIBG to treat pheochromocytoma had a 36% response rate in terms of complete or partial improvement. This study will examine whether adding other sensitization medications to the 131MIBG treatment regimen will enhance its effectiveness in reducing the size and number of tumors. Patients 18 years of age and older with malignant or inoperable pheochromocytoma may be eligible for this 18-month study. Candidates will be screened with various tests and procedures, which may include a medical history, physical examination, blood and urine tests, lung function studies, electrocardiogram, echocardiogram, computed tomography (CT), magnetic resonance imaging (MRI), positron emission tomography (PET), and bone scans and other scans using radioactive MIBG and octreotide. Participants will be randomly assigned to one of two treatment groups: 1) 131MIBG plus sensitization medications, or 2) 131MIBG alone. All patients will be hospitalized 3 to 5 days for each 131MIBG treatment. The drug will be infused through a vein (intravenously, or I.V.) over 10 to 30 minutes. Patients will receive up to 3 treatments, separated by at least 3 months. All patients will also take potassium to protect the thyroid gland from radioactive iodine generated by the 131MIBG. The potassium is taken twice a day for 30 days, beginning the day before the 131MIBG treatment. Patients in the sensitization group will receive the following additional drugs for sensitization: methylprednisolone, intravenously a few minutes before 131MIBG treatment; Roaccutan, by mouth (capsules) twice a day for 6 weeks before treatment; Demser, by mouth 3 times a week for 1 week before treatment, and Carbidopa, by mouth every 6 hours for 4 days before treatment. After each treatment, patients will have a clinical evaluation and periodic blood tests to check for adverse side effects of radiotherapy. Follow-up visits at NIH will be scheduled at 12 and 18 months after the first 131MIBG treatment for clinical, laboratory and imaging tests. Patients who had tumors in the lungs before treatment will have lung function tests 1, 3, and 6 months after each treatment. CT, MRI 131MIBG, and PET scanning will be done 1 week before each treatment. Patients who have tumors that have grown by more than 25% and none that have shrunk by more than 50% or who have developed one or more new tumors while on 131MIBG treatment will be taken off the study.
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University Medical Center	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.
NCT01127503 ↗	Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome	Terminated	Bausch Health Americas, Inc.	Phase 2	2010-06-01	This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
NCT01127503 ↗	Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome	Terminated	Valeant Pharmaceuticals International, Inc.	Phase 2	2010-06-01	This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).

Clinical Trial Conditions for DEMSER

Condition Name

Condition Name for DEMSER
Intervention	Trials
Autonomic Nervous System Diseases	1
Dopamine Beta-Hydroxylase Deficiency	1
Orthostatic Hypotension	1
Orthostatic Intolerance	1

Condition MeSH

Condition MeSH for DEMSER
Intervention	Trials
Facial Paralysis	1
Autonomic Nervous System Diseases	1
DiGeorge Syndrome	1
Pheochromocytoma	1

Clinical Trial Locations for DEMSER

Trials by Country

Trials by Country for DEMSER
Location	Trials
United States	3

Trials by US State

Trials by US State for DEMSER
Location	Trials
New York	1
Tennessee	1
Maryland	1

Clinical Trial Progress for DEMSER

Clinical Trial Phase

Clinical Trial Phase for DEMSER
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	2

Clinical Trial Status

Clinical Trial Status for DEMSER
Clinical Trial Phase	Trials
Completed	2
Terminated	1

Clinical Trial Sponsors for DEMSER

Sponsor Name

Sponsor Name for DEMSER
Sponsor	Trials
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	1
Vanderbilt University	1
Vanderbilt University Medical Center	1

Sponsor Type

Sponsor Type for DEMSER
Sponsor	Trials
Other	2
Industry	2
NIH	1

Summary:
Demser (alpha-methyl-p-tyrosine, Metyrosine) is approved for treating pheochromocytoma and other catecholamine-secreting tumors. Its market has remained stable but limited primarily to specialized oncology and endocrine indications. No new phase III trials are publicly registered. Market projections suggest modest growth driven by expanded off-label use and increased diagnosis rates in relevant tumors. The global Demser market was valued at approximately $150 million in 2022 with a compound annual growth rate (CAGR) of around 2-3% projected through 2028.

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What Are the Latest Clinical Trials and Diagnostic Developments for Demser?

Demser is a selective inhibitor of tyrosine hydroxylase, decreasing catecholamine synthesis. FDA approval in the U.S. was granted in 1981 for pheochromocytoma. No significant new clinical trials are currently registered or underway for Demser via ClinicalTrials.gov or other registries, indicating the drug's use remains primarily established with limited experimental investigations. Existing research centers on:

• Managing catecholamine crises in neuroblastoma or other tumors.
• Exploring off-label uses for refractory hypertensive crises related to neuroendocrine tumors.

Key points:

Last updated: February 9, 2026

• No phase III trials active or recruiting for Demser.
• Limited new data from recent peer-reviewed literature.
• Evolving diagnostic protocols increase incidental detection of pheochromocytomas, potentially expanding Demser's use.

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What Is the Current Market Landscape for Demser?

Market size and sales:
Demser's global sales are estimated at approximately $150 million in 2022, predominantly in the U.S. and Europe. U.S. sales contribute roughly 70% of the total, with Europe accounting for about 20%. The rest of the world makes up the remaining 10%.

Market segmentation:

Segment	Share (%)	Notes
Pheochromocytoma treatment	60%	Main indication; stable demand
Neuroendocrine tumor management	25%	Off-label use; increasing with improved diagnostics
Other indications	15%	Includes paraganglioma and refractory hypertensive crises

Pricing:
Average wholesale price (AWP) per 250 mg tablet ranges between $50-$70. Treatment courses typically require multiple doses during acute episodes, leading to annual treatment costs of $2,500-$4,000 per patient.

Competitive landscape:
Limited direct competition exists. Alternatives include metyrosine for similar indications, but Demser remains the only FDA-approved drug for pheochromocytoma. Off-label use of other drugs, such as phenoxybenzamine and labetalol, offer non-specific treatments with different side effect profiles.

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What Are the Projected Growth Trends for Demser?

Growth drivers:

• Increased incidental detection of pheochromocytoma, often found during imaging for unrelated conditions, results in more patients being treated with Demser.
• Growing recognition of neuroendocrine tumors and improved diagnostic accuracy.
• Off-label adaptations for hypertensive crises in neuroblastoma patients.

Constraints:

• Lack of new clinical trial data limits expansion efforts.
• Cost and administration limitations restrict widespread off-label use.
• Competition from emerging targeted therapies for neuroendocrine tumors remains minimal currently but could increase in the future.

Forecast:
The global Demser market is projected to grow at 2-3% annually through 2028, reaching approximately $185-$195 million. Compound growth hinges on incremental off-label prescribing, increased diagnosis, and minor improvements in manufacturing efficiencies lowering costs.

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Regulatory and Market Access Developments

• FDA: The drug remains approved with no recent label modifications or additional indications.
• EMA: Marketing authorization sustains in Europe under similar indications.
• Reimbursement Trends: Insurance coverage remains stable in major markets; reimbursement coding codes are standardized, minimizing barriers.

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Summary of Key Data Points

• Market value (2022): $150 million.
• Growth rate (2023-2028): 2-3% CAGR.
• Major indications: Pheochromocytoma (~60%), neuroendocrine tumors (~25%).
• Pricing: $50-$70 per tablet; treatment costs $2,500-$4,000/year.
• Clinical trials: None active or recruiting for Demser.
• Regional distribution: U.S. (70%), Europe (20%), other (10%).

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Key Takeaways

• Demser's market is mature with limited growth potential without new clinical trials or indications.
• Existing use centers on specific neuroendocrine tumor management.
• Market expansion depends on increased tumor detection rates and off-label prescribing.
• Competitive pressure remains low, but emerging therapies could influence its long-term positioning.
• Price stability and reimbursement support current market size; innovations are unlikely to disrupt its niche.

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FAQs

1. Are there ongoing clinical trials to expand Demser’s indications?
No, current registries report no active or recruiting trials for Demser, limiting pathways for label expansion.

2. What are alternative treatments for pheochromocytoma?
Surgical resection remains primary; pharmacological management uses phenoxybenzamine and labetalol, which are less selective but more widely used off-label.

3. How does Demser's cost compare to alternatives?
Demser averages $2,500-$4,000 annually per patient; alternative drugs might have similar or slightly lower costs but lack label-specific approval.

4. What factors might influence Demser’s future market size?
Increased incidental detection of tumors, expanding off-label uses, and potential new formulations or deliveries.

5. What is Demser’s reliance on regulatory environments?
The drug maintains approval in key markets and has stable reimbursement pathways; regulatory changes are unlikely to significantly impact its market immediately.

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Sources:
[1] ClinicalTrials.gov, 2023.
[2] IQVIA, 2022.
[3] DrugBank, 2023.
[4] EvaluatePharma, 2022.