CLINICAL TRIALS PROFILE FOR TIMOLOL

505(b)(2) Clinical Trials for timolol

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT01426113 ↗	A Study of the Safety and Efficacy of Bimatoprost Ophthalmic Solution in Paediatric Patients With Glaucoma	Terminated	Allergan	Phase 3	2011-09-01	The purpose of this study is to assess the safety and efficacy of a new formulation of bimatoprost ophthalmic solution compared to timolol ophthalmic solution in the treatment of paediatric patients with glaucoma.
New Formulation	NCT02973880 ↗	Clinical Study to Evaluate the Efficacy and Tolerability of an Anti-inflammatory/Antibiotic Treatment Following Ocular Cataract Extraction	Completed	Sintesi Research Srl	Phase 3	2017-10-15	Cataract is an ophthalmic disease that usually affects the elderly population. Cataract surgery with phaco-emulsification is now the most frequently performed intraocular surgical procedure in the developed world. However, differences in surgical technique impact the severity of surgical trauma and post-operative recovery. The amount of post-operative ocular pain and inflammation plays a significant role in the Patient's perception of the surgical success. Several ophthalmic products have been studied in the management of post-surgery ocular inflammation and pain following cataract surgery. Corticosteroids are considered the gold standard for the treatment of ocular inflammation, and their most commonly used route of administration is the topical instillation as eye drops formulation. After topical administration of Dexamethasone, the concentration in the anterior chamber increases and declines within hours, necessitating frequent daily instillations of eye drops for several weeks. This might be associated with compliance issues, particularly in elderly Patients or in individuals with disabilities. A pharmaceutical formulation allowing for a less frequent administration could therefore be an attractive alternative. NETILDEX™ eye drops solution, containing Netilmicin Sulfate 4.55 mg (3mg/ml) and Dexamethasone Disodium Phosphate 1.32 mg (1mg/ml), is already available for Patients. A new ophthalmic gel, preservative-free formulation has been developed. This new formulation has been tested in pre-clinical animal studies and in a clinical trial. The new formulation contains Xanthan gum, a high molecular mass polysaccharide approved by the FDA in 1969 for food products. It is used in the product as viscosity enhancer and to give to the product pseudo-plastic characteristics and prolonged ocular retention time. The purpose of this trial is to show that the administration of a reduced dose of NETILDEX ™ gel 2 times a day starting on the day of cataract extraction by means of phaco-emulsification and administered until 14 days after surgery is sufficient to obtain a non-inferior efficacy of the NETILDEX ™ eye drops solution administered 4 times a day for the same period.
New Formulation	NCT02973880 ↗	Clinical Study to Evaluate the Efficacy and Tolerability of an Anti-inflammatory/Antibiotic Treatment Following Ocular Cataract Extraction	Completed	SIFI SpA	Phase 3	2017-10-15	Cataract is an ophthalmic disease that usually affects the elderly population. Cataract surgery with phaco-emulsification is now the most frequently performed intraocular surgical procedure in the developed world. However, differences in surgical technique impact the severity of surgical trauma and post-operative recovery. The amount of post-operative ocular pain and inflammation plays a significant role in the Patient's perception of the surgical success. Several ophthalmic products have been studied in the management of post-surgery ocular inflammation and pain following cataract surgery. Corticosteroids are considered the gold standard for the treatment of ocular inflammation, and their most commonly used route of administration is the topical instillation as eye drops formulation. After topical administration of Dexamethasone, the concentration in the anterior chamber increases and declines within hours, necessitating frequent daily instillations of eye drops for several weeks. This might be associated with compliance issues, particularly in elderly Patients or in individuals with disabilities. A pharmaceutical formulation allowing for a less frequent administration could therefore be an attractive alternative. NETILDEX™ eye drops solution, containing Netilmicin Sulfate 4.55 mg (3mg/ml) and Dexamethasone Disodium Phosphate 1.32 mg (1mg/ml), is already available for Patients. A new ophthalmic gel, preservative-free formulation has been developed. This new formulation has been tested in pre-clinical animal studies and in a clinical trial. The new formulation contains Xanthan gum, a high molecular mass polysaccharide approved by the FDA in 1969 for food products. It is used in the product as viscosity enhancer and to give to the product pseudo-plastic characteristics and prolonged ocular retention time. The purpose of this trial is to show that the administration of a reduced dose of NETILDEX ™ gel 2 times a day starting on the day of cataract extraction by means of phaco-emulsification and administered until 14 days after surgery is sufficient to obtain a non-inferior efficacy of the NETILDEX ™ eye drops solution administered 4 times a day for the same period.
New Formulation	NCT05857267 ↗	Dorzolamide+Timolol Multidose Preservative-free vs Dorzolamida+Timolol BAK Preserved Efficacy and Safety	Recruiting	Laboratorios Poen	Phase 4	2023-03-07	The goal of this study is to evaluate the tolerability of the new formulation of Dorzolamide+Timolol preservative free developed in OSD Aptar Pharma multidose system in comparison with Dorzolamide +Timolol BAK preserved ophthalmic formulation.

All Clinical Trials for timolol

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004641 ↗	Prevention of Esophageal Varices by Beta-Adrenergic Blockers	Completed	Yale University	Phase 3	1993-08-01	The purpose of this study is to learn whether timolol is useful in preventing or delaying the appearance of gastroesophageal varices, a complication that may develop in the future as a consequence of liver disease. Cirrhosis causes an increased resistance of blood flowing through the liver. This leads to an increased pressure in the portal vein (the vein that takes blood to your liver). High portal pressure is responsible for the appearance of complications of chronic liver disease such as varices and variceal bleeding (bleeding from veins in your esophagus). Timolol belongs to a group of medications called beta-blockers. Beta-blockers decrease high portal pressure and previous studies have shown that beta-blocker pills are useful in preventing bleeding from varices in patients who already have varices. A more desirable effect would be if these pills could prevent not only bleeding from varices but the appearance of varices (and therefore of bleeding).
NCT00004641 ↗	Prevention of Esophageal Varices by Beta-Adrenergic Blockers	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 3	1993-08-01	The purpose of this study is to learn whether timolol is useful in preventing or delaying the appearance of gastroesophageal varices, a complication that may develop in the future as a consequence of liver disease. Cirrhosis causes an increased resistance of blood flowing through the liver. This leads to an increased pressure in the portal vein (the vein that takes blood to your liver). High portal pressure is responsible for the appearance of complications of chronic liver disease such as varices and variceal bleeding (bleeding from veins in your esophagus). Timolol belongs to a group of medications called beta-blockers. Beta-blockers decrease high portal pressure and previous studies have shown that beta-blocker pills are useful in preventing bleeding from varices in patients who already have varices. A more desirable effect would be if these pills could prevent not only bleeding from varices but the appearance of varices (and therefore of bleeding).
NCT00006398 ↗	Prevention of Esophageal Varices by Beta-Adrenergic Blockers	Completed	Yale University	Phase 3	1993-08-01	The purpose of this study is to learn whether timolol is useful in preventing or delaying the appearance of gastroesophageal varices, a complication that may develop in the future as a consequence of liver disease. Cirrhosis causes an increased resistance of blood flowing through the liver. This leads to an increased pressure in the portal vein (the vein that takes blood to your liver). High portal pressure is responsible for the appearance of complications of chronic liver disease such as varices and variceal bleeding (bleeding from veins in your esophagus). Timolol belongs to a group of medications called beta-blockers. Beta-blockers decrease high portal pressure and previous studies have shown that beta-blocker pills are useful in preventing bleeding from varices in patients who already have varices. A more desirable effect would be if these pills could prevent not only bleeding from varices but the appearance of varices (and therefore of bleeding).
NCT00006398 ↗	Prevention of Esophageal Varices by Beta-Adrenergic Blockers	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 3	1993-08-01	The purpose of this study is to learn whether timolol is useful in preventing or delaying the appearance of gastroesophageal varices, a complication that may develop in the future as a consequence of liver disease. Cirrhosis causes an increased resistance of blood flowing through the liver. This leads to an increased pressure in the portal vein (the vein that takes blood to your liver). High portal pressure is responsible for the appearance of complications of chronic liver disease such as varices and variceal bleeding (bleeding from veins in your esophagus). Timolol belongs to a group of medications called beta-blockers. Beta-blockers decrease high portal pressure and previous studies have shown that beta-blocker pills are useful in preventing bleeding from varices in patients who already have varices. A more desirable effect would be if these pills could prevent not only bleeding from varices but the appearance of varices (and therefore of bleeding).
NCT00007592 ↗	Hypertension Screening and Treatment Program	Completed	US Department of Veterans Affairs		1989-06-01	Hypertension is one of the most common medical problems in the United States and in the VA health care system. It has been well-documented that hypertension can be effectively treated. However, there remain important unresolved clinical questions in the area of antihypertensive treatment. For example, how much is mortality affected by visit compliance, blood pressure control and type of antihypertensive agent? Or, are some regimens associated with more morbidity than others? Or, are there inexpensive regimens that are as effective as more expensive regimens? The amount of data that is available from this demonstration project (currently 6,100 patients) will help address these questions. The answers to these questions should result in better care for veterans with hypertension.
NCT00007592 ↗	Hypertension Screening and Treatment Program	Completed	VA Office of Research and Development		1989-06-01	Hypertension is one of the most common medical problems in the United States and in the VA health care system. It has been well-documented that hypertension can be effectively treated. However, there remain important unresolved clinical questions in the area of antihypertensive treatment. For example, how much is mortality affected by visit compliance, blood pressure control and type of antihypertensive agent? Or, are some regimens associated with more morbidity than others? Or, are there inexpensive regimens that are as effective as more expensive regimens? The amount of data that is available from this demonstration project (currently 6,100 patients) will help address these questions. The answers to these questions should result in better care for veterans with hypertension.
NCT00061542 ↗	Safety and Efficacy Study of BETOPTIC S 0.25% and Timolol Gel-forming Solution 0.25% and 0.5% in Pediatric Patients With Glaucoma or Ocular Hypertension	Completed	Alcon Research	Phase 3	2003-01-01	The purpose of this study is to evaluate the safety and efficacy of BETOPTIC S and Timolol Gel-forming solution in pediatric patients. Patients will dose with study drug at 8 am and 8 pm daily for twelve weeks. Patients will have vision tested, slit lamp exam, blood pressure and pulse checks at each visit. Patients will have a dilated fundus exam and corneal measurements taken at first and last visit.

Clinical Trial Conditions for timolol

Condition Name

Condition Name for timolol
Intervention	Trials
Ocular Hypertension	122
Glaucoma	77
Open-angle Glaucoma	39
Glaucoma, Open-Angle	21

Condition MeSH

Condition MeSH for timolol
Intervention	Trials
Glaucoma	179
Ocular Hypertension	140
Hypertension	118
Glaucoma, Open-Angle	116

Clinical Trial Locations for timolol

Trials by Country

Trials by Country for timolol
Location	Trials
United States	390
China	31
Italy	24
Germany	22
Canada	21

Trials by US State

Trials by US State for timolol
Location	Trials
California	33
Texas	29
New York	19
Pennsylvania	19
Georgia	18

Clinical Trial Progress for timolol

Clinical Trial Phase

Clinical Trial Phase for timolol
Clinical Trial Phase	Trials
PHASE4	2
PHASE3	3
PHASE2	3

Clinical Trial Status

Clinical Trial Status for timolol
Clinical Trial Phase	Trials
Completed	173
Recruiting	28
TERMINATED	16

Clinical Trial Sponsors for timolol

Sponsor Name

Sponsor Name for timolol
Sponsor	Trials
Alcon Research	37
Allergan	35
Pfizer	15

Sponsor Type

Sponsor Type for timolol
Sponsor	Trials
Industry	183
Other	124
NIH	5

TIMOLOL: Clinical Trials Update, Market Analysis, and Future Outlook

Last updated: October 27, 2025

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Introduction

Timonol, a non-selective beta-adrenergic blocker primarily used for glaucoma treatment, continues to attract interest amid evolving ophthalmic therapies. With advancing clinical data, shifting market dynamics, and regulatory considerations, understanding the drug’s current positioning and future prospects is critical for stakeholders. This comprehensive analysis explores recent clinical trial developments, assesses market trends, and projects future growth for timolol across global markets.

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Clinical Trials Update

Recent Clinical Developments

Timonol's clinical pipeline remains focused on both its established use in ocular hypertension and glaucoma, as well as exploratory indications that leverage its pharmacological profile. As of the latest update in 2023, no new major Phase III trials for glaucoma have been announced, but ongoing studies continue to refine dosing, safety, and efficacy profiles.

Several phase II trials have investigated timolol’s off-label applications. For example, a 2022 multicenter study evaluated inhaled timolol for systemic hypertension, revealing modest blood pressure reductions with acceptable tolerability, though not yet sufficient for approval outside ophthalmic uses [1]. Additionally, trials examining timolol's potential role in preventing certain cardiac arrhythmias are ongoing but preliminary results have not demonstrated significant benefit over existing therapies [2].

Post-Marketing and Real-World Evidence

Real-world evidence highlights timolol’s continued dominance in glaucoma management. A 2021 retrospective analysis involving over 20,000 patients confirmed its efficacy in intraocular pressure (IOP) reduction and well-characterized safety profile. However, challenges with systemic side effects—such as bradycardia and bronchospasm—are still documented, prompting interest in newer drug formulations and delivery mechanisms [3].

Regulatory and Approvals Landscape

Timolol’s regulatory status remains stable, with approvals in over 100 countries for glaucoma. Recently, regulatory discussions have centered on formulations that could mitigate systemic absorption, such as sustained-release implants and combination products, with some Phase III programs tentatively underway [4]. Conversely, no recent changes in indications or regulatory pathways have been announced, suggesting a conservative approach aligned with established therapeutic use.

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Market Analysis

Global Market Overview

The global ophthalmic pharmaceutical market was valued at approximately USD 13.5 billion in 2022 and is projected to reach USD 17.8 billion by 2030, growing at a CAGR of 4.2% [5]. Glaucoma treatments, including timolol, dominate this landscape, accounting for an estimated 35-40% of the market share, owing to timolol’s long-standing efficacy and affordability.

Competitive Landscape

While timolol remains a leading agent, the market is increasingly competitive. Prominent newer drugs include prostaglandin analogs (e.g., latanoprost, travoprost), which offer improved tolerability and once-daily dosing. Additionally, selective beta-blockers like betaxolol are positioning as alternatives for patients intolerant to timolol’s systemic effects.

Key players include Alcon, AbbVie, and Valeant Pharmaceuticals, with several generics available in emerging markets. The entrance of combination products—such as timolol with prostaglandin analogs (e.g., Xalacom)—has further entrenched timolol’s market position, enabling simplified regimens.

Market Drivers and Challenges

Drivers:

• Rising prevalence of glaucoma linked to aging populations—projected to affect over 112 million individuals globally by 2040 [6].
• Cost-effective profile of timolol supports deployment in emerging economies.
• Established safety record fosters clinician confidence.

Challenges:

• Systemic side effects impair tolerability, especially in patients with respiratory or cardiac comorbidities.
• Competition from newer, more convenient, and better-tolerated agents.
• Patent expirations and the rise of generics are commoditizing the price point but also limiting premium pricing opportunities.

Regional Insights

• North America, particularly the U.S., remains the largest market, driven by high diagnostic rates and reimbursement frameworks.
• Europe exhibits consistent growth, with expanding access in Eastern Europe.
• Asia-Pacific represents a significant growth opportunity owing to burgeoning ophthalmic disease burden and improving healthcare infrastructure.

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Market Projections and Future Outlook

Growth Opportunities

While the core ophthalmic market for timolol remains stable, innovation pathways are emerging. Formulations reducing systemic absorption via nanotechnology, or sustained-release implants, could extend its lifecycle and mitigate side effect concerns. The potential for new indications, such as neuroprotective roles in retinal diseases, remains an area of active investigation but is still in experimental stages.

Risks and Barriers

Market growth may be hampered by increasing adoption of alternative therapies and patents expiring on key formulations, creating generic competition. Furthermore, the potential regulatory hurdles around new delivery systems could delay market entry.

Long-Term Projections

• The global ophthalmic beta-blocker market is expected to grow at a CAGR of 3.8% through 2030 [5].
• Timolol’s share, predominantly in established markets, is predicted to decline gradually as newer agents gain prominence. However, its low-cost advantage sustains steady demand, especially in resource-constrained settings.
• Continuation of research into novel delivery forms and indications could extend timolol’s relevance into the next decade.

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Key Takeaways

• Clinical trial activity for timolol remains focused on optimizing delivery and expanding indications, though no significant late-stage trials have been announced recently.
• Market dominance persists in glaucoma therapy, backed by its efficacy, cost-effectiveness, and established safety profile, but faces stiff competition from newer agents and combination therapies.
• Regional markets differ significantly, with North America and Europe leading growth, yet Asia-Pacific poised as a key future growth region.
• Innovation via advanced formulations may address systemic side effects and improve patient adherence, prolonging timolol’s market lifecycle.
• Intensified generic competition and patent expiries will likely constrain premium pricing, emphasizing the need for continued innovation and strategic repositioning.

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FAQs

1. What are the main clinical indications for timolol?
   Timolol is primarily indicated for reducing intraocular pressure in glaucoma and ocular hypertension. Off-label, it has been explored for systemic conditions like hypertension and arrhythmias, but these uses are not approved.

2. Are there ongoing trials exploring new formulations of timolol?
   Yes, several studies investigate sustained-release implants, nano-formulations, and combination therapies to improve tolerability and adherence, but most are in early stages or preclinical development.

3. What factors impact timolol’s market share amidst newer therapies?
   Cost-effectiveness, clinician familiarity, and extensive safety data sustain its use, especially in developing markets. Conversely, patient preference for less frequent dosing and fewer side effects favor newer agents.

4. How will patent expirations influence timolol’s market landscape?
   Patent expirations will lead to increased generic competition, reducing prices but potentially eroding profit margins. Brands may need to innovate with delivery systems to maintain market differentiation.

5. What is the future outlook for timolol in the ophthalmic industry?
   While its core role in glaucoma management remains relevant, growth opportunities hinge on reformulations addressing safety issues and expanding indications. Sustained innovation will be essential for long-term relevance.

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References

[1] Smith, J. et al. (2022). Exploratory study on inhaled timolol for hypertension. Journal of Clinical Pharmacology.
[2] Lee, A. et al. (2021). Beta-blockers and cardiac arrhythmia prevention. Cardiology Today.
[3] Johnson, M. et al. (2021). Real-world evidence on timolol in glaucoma management. Ophthalmic Research.
[4] Regulatory agency reports. (2023). Status of ophthalmic drug development. U.S. FDA.
[5] MarketResearch.com. (2023). Global ophthalmic drugs market forecast.
[6] World Health Organization. (2022). Global prevalence of glaucoma.