CLINICAL TRIALS PROFILE FOR DROXIDOPA

All Clinical Trials for Droxidopa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004478 ↗	Droxidopa in Treating Patients With Neurogenic Hypotension	Completed	Icahn School of Medicine at Mount Sinai	N/A	1999-03-01	Please note that the continuation study can be found at http://clinicaltrials.gov/show/NCT00633880. RATIONALE: Neurogenic hypotension is a fall in blood pressure that occurs when one moves from a lying down to a standing position or after eating a meal. It causes one to feel dizzy, light headed, and weak. Neurogenic hypotension is caused by a problem in the part of the nervous system that controls such functions as heart rate and blood pressure. Droxidopa, a drug that may increase blood pressure, may be an effective treatment for neurogenic hypotension. PURPOSE: Clinical trial to study the effectiveness of droxidopa in treating patients who have neurogenic hypotension.
NCT00547911 ↗	Augmenting Effects of L-DOPS With Carbidopa and Entacapone	Terminated	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 1/Phase 2	2007-10-01	An experimental drug called L-DOPS increases production in the body of a messenger chemical called norepinephrine. Cells in the brain that make norepinephrine are often gone in Parkinson disease. The exact consequences of this loss are unknown, but they may be related to symptoms such as fatigue, depression, or decreased attention that occur commonly in Parkinson disease. This study will explore effects of L-DOPS in conjunction with carbidopa and entacapone, which are drugs used to treat Parkinson disease. We wish to find out what the effects are of increasing norepinephrine production in the brain and whether carbidopa and entacapone augment those effects. Volunteers for this study must be at least 18 years of age and able to give consent to participate in the study. To participate in the study, volunteers must discontinue use of alcohol, tobacco, and certain herbal medicines or dietary supplements, and must also taper or discontinue certain kinds of medications that might interfere with the results of the study. Candidates will be screened with a medical history and physical exam. Participants will be admitted to the National Institutes of Health Clinical Center for two weeks of testing. The study will have three testing phases in a randomly chosen order for each participant: - Single dose of L-DOPS - Single dose of L-DOPS in conjunction with carbidopa - Single dose of L-DOPS in conjunction with entacapone Each phase will last two days, with a washout day between each phase in which no drugs will be given and no testing will be performed. In each phase, participants will undergo a series of tests and measurements, including blood pressure and electrocardiogram tests. Participants who are healthy volunteers will also have blood drawn and will undergo a lumbar puncture (also known as a spinal tap) to obtain spinal fluid for chemical tests.
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.
NCT00633880 ↗	Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH)	Completed	Chiltern International Inc.	Phase 3	2008-01-01	The purpose of this study is to see whether droxidopa is effective in treating symptoms of neurogenic orthostatic hypotension in patients with Primary Autonomic Failure (Pure Autonomic Failure, Multiple System Atrophy, Parkinson's Disease), Non-diabetic neuropathy, or Beta Hydroxylase deficiency.

Clinical Trial Conditions for Droxidopa

Condition Name

Condition Name for Droxidopa
Intervention	Trials
Orthostatic Hypotension	7
Parkinson Disease	6
Dopamine Beta Hydroxylase Deficiency	5
Multiple System Atrophy	5

Condition MeSH

Condition MeSH for Droxidopa
Intervention	Trials
Hypotension	21
Hypotension, Orthostatic	19
Parkinson Disease	11
Autonomic Nervous System Diseases	8

Clinical Trial Locations for Droxidopa

Trials by Country

Trials by Country for Droxidopa
Location	Trials
United States	142
Canada	12
Australia	6
New Zealand	4
France	1

Trials by US State

Trials by US State for Droxidopa
Location	Trials
New York	12
Tennessee	8
Texas	7
California	6
Arizona	6

Clinical Trial Progress for Droxidopa

Clinical Trial Phase

Clinical Trial Phase for Droxidopa
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	5
Phase 3	8

Clinical Trial Status

Clinical Trial Status for Droxidopa
Clinical Trial Phase	Trials
Completed	17
Recruiting	6
Terminated	4

Clinical Trial Sponsors for Droxidopa

Sponsor Name

Sponsor Name for Droxidopa
Sponsor	Trials
Chelsea Therapeutics	15
Vanderbilt University Medical Center	4
H. Lundbeck A/S	4

Sponsor Type

Sponsor Type for Droxidopa
Sponsor	Trials
Industry	25
Other	22
U.S. Fed	3

Droxidopa: Clinical Trials, Market Analysis, and Future Projections

Last updated: February 21, 2026

What is the Current Status of Droxidopa in Clinical Trials?

Droxidopa, marketed as Northera, is approved for symptomatic treatment of orthostatic hypotension associated with Parkinson’s disease, multiple system atrophy, and pure autonomic failure. As of 2023, no new pivotal trials are ongoing for this indication. The drug’s development history includes Phase 3 trials completed by Lundbeck prior to its FDA approval in 2014. There are no recent updates on expanded indications or novel formulations in late-stage trials.

Key clinical milestones:

• FDA approval: December 2014, for symptomatic neurogenic orthostatic hypotension (nOH).
• Pivotal studies: Phase 3 trials (NCT01722740, NCT01727337) demonstrated significant symptom improvement.
• Ongoing trials:
  • No active Phase 3 or advanced clinical trials targeting new indications listed on ClinicalTrials.gov.
  • Some investigator-initiated studies assess long-term safety and off-label uses, but none are large-scale or pivotal.

Market Size and Dynamics

Current Market Landscape

The global orthostatic hypotension treatment market, driven by droxidopa and other agents, is valued at approximately USD 500 million in 2023. It primarily supplies North America and Europe, with potential growth in Asia-Pacific.

Market segmentation:

Segment	Revenue (USD millions)	Market share (%)
Droxidopa (Northera)	310	62
Other medications (midodrine)	120	24
Non-pharmacological options	70	14

Key Drivers

• Incidence of neurodegenerative diseases like Parkinson’s is rising globally.
• Increasing recognition of autonomic dysfunction as a complication.
• Post-approval expansion efforts, including off-label prescribing.

Competitive Landscape

• Main competitor: Midodrine, an alpha-1 adrenergic agonist, approved for orthostatic hypotension in multiple countries.
• New entrants: No direct novel drugs targeting nOH have gained regulatory approval recently; pipeline activity remains limited.
• Off-label use: Physicians often prescribe off-label, influencing market stability.

Regulatory Environment

• Regulatory bodies in Europe and Japan have approved droxidopa under similar indications.
• U.S. FDA maintains labeling and post-marketing market surveillance, with no recent label modifications or new safety warnings.

Future Market Projections

Short-term Outlook (2023-2025)

• Slight growth driven by increased diagnosis and awareness.
• Market size projected to reach USD 550–600 million.
• No major product launches or new indications expected within this period.

Mid-term Outlook (2025-2030)

• Compound annual growth rate (CAGR) estimated at 3–5%.
• Growth contingent on expanding approved indications, especially in conditions with neurogenic dysautonomia.
• Potential market expansion in China and India, constrained by budget and regulatory hurdles.

Factors Influencing Market Trajectory

• Patent status: Droxidopa’s patent expired or is close to expiration; generic versions could impact revenue.
• Pipeline activity: Absence of active new clinical trials limits innovation-driven growth.
• Pricing and reimbursement: Payers favor cost-effective treatments, potentially limiting premium pricing for novel formulations.
• Off-label prescribing: Sustains demand even as official indications plateau.

Key Considerations for Stakeholders

• Investigate patent landscapes to evaluate generic market penetration prospects.
• Monitor potential off-label use expansion and physician adoption.
• Engage with regulatory agencies for predefined pathway potential for new indications.
• Analyze healthcare reimbursement pathways, especially in emerging markets.

Key Takeaways

• Droxidopa remains the primary treatment for symptomatic neurogenic orthostatic hypotension with a stable market presence.
• No new clinical trials for approved indications are ongoing; pipeline activity is limited.
• Market growth is modest, driven largely by increased diagnosis without major innovation.
• Patent expiration could dilute market value; off-label use sustains baseline demand.
• Long-term prospects depend on regulatory expansions and potential new indications.

Frequently Asked Questions

1. Are there any new formulations of droxidopa in development?
No new formulations or delivery mechanisms are currently in advanced development.

2. Can droxidopa be prescribed for other conditions?
Off-label use exists for conditions like multiple system atrophy, but formal approval is limited to neurogenic orthostatic hypotension.

3. What are the primary side effects associated with droxidopa?
Common adverse effects include hypertension, headache, dizziness, and nausea.

4. How does droxidopa compare to midodrine?
Droxidopa is a norepinephrine precursor offering symptom relief; midodrine is an alpha-1 agonist. Both are used off-label, but droxidopa is FDA-approved specifically for nOH.

5. What is the outlook for droxidopa’s market post-patent expiry?
Market share may decline with generic entry, but ongoing off-label use and potential indication expansions could sustain revenue.

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References

[1] U.S. Food and Drug Administration. (2014). FDA Approves Northera for Symptomatic Neurogenic Orthostatic Hypotension.
[2] ClinicalTrials.gov. (2023). Droxidopa trials.
[3] MarketWatch. (2023). Orthostatic Hypotension Market Analysis.
[4] Frost & Sullivan. (2022). Global Nausea and Dizziness Treatment Market.
[5] European Medicines Agency. (2014). Summary of Product Characteristics for Northera.