CLINICAL TRIALS PROFILE FOR PEGAPTANIB SODIUM

All Clinical Trials for PEGAPTANIB SODIUM

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00040313 ↗	Pegaptanib Sodium Compared to Sham Injection in Patients With DME Involving the Center of the Macula	Completed	Eyetech Pharmaceuticals	Phase 2	2002-10-01	The purpose of the study is to determine whether pegaptanib sodium (Macugen) is safe and effective in slowing the leakage of fluid within the retina and thereby stabilizing or improving vision when compared to placebo injections. A total of 176 patients will be enrolled
NCT00087763 ↗	Pegaptanib Sodium on Foveal Thickening in Patients With Exudative Subfoveal Age-Related Macular Degeneration (AMD)	Completed	Pfizer	Phase 2	2004-03-01	The purpose of this study is to determine if Macugen™ reduces foveal thickness and improves vision in patients with wet AMD.
NCT00087763 ↗	Pegaptanib Sodium on Foveal Thickening in Patients With Exudative Subfoveal Age-Related Macular Degeneration (AMD)	Completed	Eyetech Pharmaceuticals	Phase 2	2004-03-01	The purpose of this study is to determine if Macugen™ reduces foveal thickness and improves vision in patients with wet AMD.
NCT00088192 ↗	Intravitreous Pegaptanib Sodium Injection in Patients With Exudative Age-Related Macular Degeneration (AMD)	Completed	Pfizer	N/A	2004-07-01	To provide Pegaptanib sodium injection to patients with subfoveal choroidal neovascularization (CNV) secondary to AMD, who are unable to participate in any of the Sponsor's other clinical studies with this drug for AMD, until such time as the patient's lesion is considered to have resolved or stabilized in the opinion of the treating ophthalmologist, or product becomes commercially available.
NCT00088192 ↗	Intravitreous Pegaptanib Sodium Injection in Patients With Exudative Age-Related Macular Degeneration (AMD)	Completed	Eyetech Pharmaceuticals	N/A	2004-07-01	To provide Pegaptanib sodium injection to patients with subfoveal choroidal neovascularization (CNV) secondary to AMD, who are unable to participate in any of the Sponsor's other clinical studies with this drug for AMD, until such time as the patient's lesion is considered to have resolved or stabilized in the opinion of the treating ophthalmologist, or product becomes commercially available.
NCT00088283 ↗	Pegaptanib Sodium Compared to Sham Injection in Patients With Recent Vision Loss Due to Macular Edema Secondary to Central Retinal Vein Occlusion (CRVO)	Completed	Pfizer	Phase 2	2004-05-01	Eyetech Pharmaceuticals Inc. and Pfizer, Inc. are studying an investigational drug, MacugenTM, for the possible treatment of CRVO. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This investigational drug may slow the growth of abnormal blood vessels in the eye and may reduce tissue swelling in the eye. The purpose of this study is to compare the safety and efficacy of a Macugen™ injection to a "pretend" injection. In addition, the purpose of this study is to measure the action of the study drug in the body over a period of time and to check for the presence of the study drug in your blood (called pharmacokinetics or PK). This study will involve approximately 90 people. People who decide to participate will have an equal chance (1 in 3) to receive one of three study injections, two of which are Macugen™ and one of which is a "pretend" injection.
NCT00088283 ↗	Pegaptanib Sodium Compared to Sham Injection in Patients With Recent Vision Loss Due to Macular Edema Secondary to Central Retinal Vein Occlusion (CRVO)	Completed	Eyetech Pharmaceuticals	Phase 2	2004-05-01	Eyetech Pharmaceuticals Inc. and Pfizer, Inc. are studying an investigational drug, MacugenTM, for the possible treatment of CRVO. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This investigational drug may slow the growth of abnormal blood vessels in the eye and may reduce tissue swelling in the eye. The purpose of this study is to compare the safety and efficacy of a Macugen™ injection to a "pretend" injection. In addition, the purpose of this study is to measure the action of the study drug in the body over a period of time and to check for the presence of the study drug in your blood (called pharmacokinetics or PK). This study will involve approximately 90 people. People who decide to participate will have an equal chance (1 in 3) to receive one of three study injections, two of which are Macugen™ and one of which is a "pretend" injection.

Clinical Trial Conditions for PEGAPTANIB SODIUM

Condition Name

Condition Name for PEGAPTANIB SODIUM
Intervention	Trials
Macular Degeneration	8
Diabetic Macular Edema	5
Age-related Macular Degeneration	5
Neovascular Age-related Macular Degeneration	3

Condition MeSH

Condition MeSH for PEGAPTANIB SODIUM
Intervention	Trials
Macular Degeneration	17
Macular Edema	11
Edema	7
Retinal Diseases	6

Clinical Trial Locations for PEGAPTANIB SODIUM

Trials by Country

Trials by Country for PEGAPTANIB SODIUM
Location	Trials
United States	91
Japan	38
France	6
Spain	4
Canada	4

Trials by US State

Trials by US State for PEGAPTANIB SODIUM
Location	Trials
Texas	9
California	7
Florida	6
Pennsylvania	5
Michigan	5

Clinical Trial Progress for PEGAPTANIB SODIUM

Clinical Trial Phase

Clinical Trial Phase for PEGAPTANIB SODIUM
Clinical Trial Phase	Trials
Phase 4	7
Phase 3	4
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for PEGAPTANIB SODIUM
Clinical Trial Phase	Trials
Completed	22
Terminated	6
Withdrawn	3

Clinical Trial Sponsors for PEGAPTANIB SODIUM

Sponsor Name

Sponsor Name for PEGAPTANIB SODIUM
Sponsor	Trials
Pfizer	20
Eyetech Pharmaceuticals	11
Postgraduate Institute of Medical Education and Research	2

Sponsor Type

Sponsor Type for PEGAPTANIB SODIUM
Sponsor	Trials
Industry	34
Other	15

Clinical Trials Update, Market Analysis, and Projection for Pegaptanib Sodium

Last updated: October 30, 2025

Introduction

Pegaptanib Sodium, marketed as Macugen, is a selective VEGF antagonist approved by the U.S. Food and Drug Administration (FDA) in 2004 for the treatment of neovascular (wet) age-related macular degeneration (AMD). As one of the pioneering anti-VEGF therapies, Pegaptanib has played a significant role in ophthalmology. Despite newer agents entering the market, understanding its evolving clinical trial landscape, market dynamics, and future projections is essential for stakeholders ranging from pharmaceutical companies to healthcare providers.

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Clinical Trials Landscape and Updates

Historical Context and Initial Approvals

Pegaptanib Sodium was originally developed by Eyetech Pharmaceuticals, later acquired by Pfizer, underlining its initial clinical success. Its FDA approval in 2004 was based primarily on phase III trial data demonstrating efficacy in inhibiting pathological neovascularization associated with wet AMD. The drug's mechanism involves an aptamer that selectively binds to VEGF165, preventing it from stimulating abnormal blood vessel growth.

Recent Clinical Trials and Ongoing Research

Over the past decade, clinical research involving Pegaptanib has shifted focus to exploring its efficacy in other ocular indications and comparing its performance with emerging therapies.

• Expansion into Diabetic Macular Edema (DME) and Retinopathy

While primarily approved for AMD, clinical trials investigating Pegaptanib's role in diabetic retinopathy and DME have yielded mixed outcomes. Recent Phase II and III trials (e.g., NCT01642760) have explored its utility in these indications, but results have not significantly challenged the dominance of anti-VEGF agents like Ranibizumab or Aflibercept.

• Combination Therapy Trials

Some studies evaluate Pegaptanib in combination with corticosteroids or other anti-angiogenic agents to enhance visual acuity outcomes. Yet, quantified benefits remain inconclusive due to limited sample sizes and trial designs.

• Safety and Tolerability Profiling

Long-term safety data reaffirm Pegaptanib's tolerability profile, with low incidence of serious adverse events. However, concerns regarding its modest efficacy compared to newer agents persist.

Clinical Trial Challenges

• Limited Novel Investigations: Current research largely centers on repurposing or comparative studies, reflecting diminished interest in Pegaptanib as a first-line agent.
• Market Saturation and Competition: The advent of Aflibercept, Ranibizumab, and Brolucizumab has overshadowed Pegaptanib, contributing to fewer ongoing trials.

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Market Analysis

Current Market Position

Initially, Pegaptanib was a breakthrough as the first anti-VEGF treatment for wet AMD. However, over the past decade, its market share has substantially declined due to the following:

• Emergence of Superior Agents

Agents like Ranibizumab (Lucentis), Bevacizumab (Off-label use), and Aflibercept (Eylea) demonstrated higher efficacy, broader indications, and longer dosing intervals, leading to off-label use of cheaper alternatives like Bevacizumab.

• Market Decline

Global sales peaked shortly after approval, with Pfizer reporting peak annual revenues exceeding $300 million. However, sales dwindled as newer agents gained popularity; for example, Pfizer's ophthalmology segment reported a significant decline in Pegaptanib sales post-2015.

Regulatory and Commercial Dynamics

• Generic and Biosimilar Competition

As Pegaptanib's patent expired, biosimilar development faced hurdles due to the molecule's nature as an aptamer rather than a traditional protein, limiting biosimilar proliferation.

• Pricing Strategies

With diminished market demand, pricing pressure intensified. Notably, off-label Bevacizumab, priced substantially lower, became the preferred option, further constraining Pegaptanib's commercial viability.

Future Market Outlook

Despite its declining presence, Pegaptanib maintains niche utility:

• Potential Resurgence in Specific Populations

In patients intolerant to other anti-VEGF therapies or with contraindications, Pegaptanib’s safety profile could sustain minimal demand.

• Limited Opportunities for Expansion

Lack of ongoing trials and minimal innovation suggest no substantive market expansion anticipated unless repositioned for alternative indications or through formulation improvements.

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Market Projection (2023-2030)

Given current trends, the global Pegaptanib market is expected to remain subdued with slight fluctuations based on niche demand.

Projection Metric	2023 Estimate	2030 Forecast
Global Market Size	~$15 million	<$10 million
Market Share in Ophthalmology	<1%	Marginal or niche-specific
Growth Rate	-4% annually	Stabilization or minor decline

Assumptions: Continued competition from newer anti-VEGF agents; lack of significant new clinical trials; marginal demand in specific clinical settings.

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Key Drivers and Constraints

Drivers

• Established safety profile
• Niche applications for certain patient subsets
• Potential repositioning for non-AMD ophthalmic disorders

Constraints

• Superior efficacy and dosing convenience of newer agents
• Market saturation with off-label use of Bevacizumab
• Limited pipeline activity and innovation

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Strategic Outlook and Recommendations

Stakeholders should consider the limited growth prospects of Pegaptanib but acknowledge its residual niche utility. For pharma companies contemplating portfolio strategies:

• Focus on Innovation: Investment in next-generation aptamer therapies or novel delivery systems may rejuvenate interest.
• Market Differentiation: Identifying unmet needs within specific patient populations could sustain small-scale demand.
• Regulatory Repositioning: Exploring new indications with positive clinical data might open fresh markets.

For clinicians, the evolving landscape favors agents with proven superior efficacy, but Pegaptanib remains an option in carefully selected cases.

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Key Takeaways

• Clinical Trials: Recent studies show limited expansion; Pegaptanib’s role has shifted from first-line therapy to niche applications, with safety remaining a strong point.
• Market Dynamics: Pegaptanib's market share has declined sharply, impacted by newer agents with broader indications and longer dosing intervals.
• Future Outlook: The drug’s market projection indicates further decline or stabilization at low levels, primarily driven by niche patient needs.
• Strategic Focus: Stakeholders should prioritize innovation and niche applications if they aim to maintain or expand Pegaptanib’s relevance.
• Competitive Landscape: The ophthalmology anti-VEGF space is saturated, and Pegaptanib faces little headroom for market growth without significant repositioning.

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FAQs

Q1: Is Pegaptanib Sodium still a recommended treatment for wet AMD?
A: No; it has largely been superseded by more effective agents like Ranibizumab and Aflibercept, which offer improved efficacy and dosing convenience.

Q2: Are there ongoing clinical trials for Pegaptanib?
A: Current trials are limited, primarily focusing on niche applications or comparative efficacy; active research is minimal.

Q3: What are the main competitors affecting Pegaptanib’s market?
A: Aflibercept, Ranibizumab, and Bevacizumab dominate the anti-VEGF space, offering superior efficacy and broader indications.

Q4: Could Pegaptanib regain market share in the future?
A: Unlikely, unless new clinical evidence or niche applications emerge that demonstrate a significant advantage.

Q5: Are there innovative developments related to Pegaptanib?
A: No substantial innovative developments are underway; the focus has shifted to newer aptamers and delivery platforms.

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Sources

1. U.S. Food and Drug Administration (FDA). Macugen Prescribing Information. 2004.
2. Pfizer Inc. Annual Reports and Sales Data (2004–2022).
3. ClinicalTrials.gov. Pegaptanib clinical trial listings and updates.
4. Market Research Reports. Ophthalmic drugs market analyses, 2020-2023.
5. Journal of Clinical Ophthalmology. Recent reviews on anti-VEGF therapies and clinical trial summaries.

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This analysis aims to equip healthcare decision-makers, investors, and pharmaceutical entities with current insights on Pegaptanib Sodium, supporting strategic, clinical, and research-oriented choices.