What Is Verteporfin?
Verteporfin is an FDA-approved medication for eye diseases. Also known by its brand name Visudyne, Verteporfin is a benzoporphyrin derivative. It functions as a photosensitizer, meaning it becomes active when exposed to light. In photodynamic therapy (PDT), Verteporfin is administered intravenously and then activated by a specific laser light, which targets and damages abnormal cells or blood vessels. This mechanism is utilized in its approved use for certain eye conditions and is being investigated for other medical challenges.
Beyond its approved indications, Verteporfin is currently being studied in clinical trials for its potential role in treating various cancers, including pancreatic cancer and glioblastoma, as well as for scar formation after skin injury. These investigational uses leverage its light-activated properties to target and treat diseased tissues.
Uses and Conditions Under Study
Verteporfin is primarily known for its approved use in treating certain eye diseases. Clinical trials involving Verteporfin have enrolled a total of 3,386 participants across 46 trials, with the first trial starting in 2002 and the latest projected to conclude in 2026. Currently, 6 trials are recruiting new participants, and 23 trials have been completed.
The majority of studies focus on retinal and choroidal conditions, where Verteporfin's photodynamic therapy (PDT) mechanism can target abnormal blood vessel growth. These include:
- Macular Degeneration (9 trials)
- Choroidal Neovascularization (8 trials)
- Polypoidal Choroidal Vasculopathy (4 trials)
- Chronic Central Serous Chorioretinopathy (2 trials)
- Neovascular Age-related Macular Degeneration (2 trials)
- Age Related Macular Degeneration (2 trials)
- Central Serous Chorioretinopathy (2 trials)
- Age-Related Macular Degeneration (2 trials)
Verteporfin is also being investigated for its potential in oncology. Its light-activated properties are being explored to treat solid tumors. Conditions under study include Stage III Pancreatic Cancer AJCC v8 (2 trials) and Stage IV Pancreatic Cancer AJCC v8 (2 trials). Additionally, it is being studied for high-grade EGFR-mutated glioblastoma and for scar formation after skin injury.
Major sponsors of these trials include industry leaders like Novartis and QLT Inc., as well as academic institutions such as Mayo Clinic and Massachusetts Eye and Ear Infirmary.
Dosing
Verteporfin is administered as an injectable solution. For its approved use in eye diseases, it is typically given as an intravenous infusion, followed by activation with a non-thermal laser light. Specific doses studied for intravitreal injection include 0.5 MG Per 0.05 ML Injection.
In clinical trials, various concentrations of Verteporfin solution have been investigated, including 0.5 mg/mL, 1.0 mg/mL, and 2.0 mg/mL. The method of administration can vary depending on the condition being studied. For instance, in some cancer trials, Verteporfin is administered intravenously approximately 60-90 minutes prior to photodynamic therapy (PDT), where optical fibers deliver light directly to the target tissue.
Investigational dosing strategies for eye conditions have explored monotherapy, combination therapy with other drugs like ranibizumab or aflibercept, and variations in light fluence (e.g., half-dose or half-fluence PDT). These studies aim to determine the most effective and safest dosages for different conditions and treatment approaches.
Side Effects
No specific side effect data for Verteporfin was provided in the clinical trial information. Therefore, a detailed list of side effects cannot be presented here.
Clinical Trial Results
Studies have investigated Verteporfin, also known by the brand name Visudyne, in combination with other treatments for age-related macular degeneration (AMD) and choroidal neovascularization (CNV).
NCT00242580: Verteporfin with Triamcinolone or Pegaptanib
A study (NCT00242580) compared Verteporfin therapy combined with either triamcinolone acetonide (at 1 mg or 4 mg doses) or pegaptanib. After 12 months:
- Vision Preservation: The highest percentage of participants who lost less than 15 letters of best corrected visual acuity (meaning their vision was maintained or improved) was 71.1% in the group treated with Verteporfin plus pegaptanib. This was compared to 63.4% in the Verteporfin plus 4 mg triamcinolone group and 59.4% in the Verteporfin plus 1 mg triamcinolone group.
- Vision Improvement (10+ Letters): A gain of 10 or more letters in visual acuity was observed in 23.7% of participants receiving Verteporfin plus pegaptanib. In comparison, 18.8% of those on Verteporfin plus 1 mg triamcinolone and 2.4% on Verteporfin plus 4 mg triamcinolone experienced this level of improvement.
- Vision Improvement (15+ Letters): A significant gain of 15 or more letters was seen in 13.2% of participants treated with Verteporfin plus pegaptanib, while 6.3% of those on Verteporfin plus 1 mg triamcinolone achieved this, and 0% in the 4 mg triamcinolone group.
- Lesion Area: The group receiving Verteporfin plus 1 mg triamcinolone showed a mean reduction in total lesion area of -0.0219 mm², while the Verteporfin plus 4 mg triamcinolone group had a mean increase of 0.1749 mm², and the Verteporfin plus pegaptanib group had a mean increase of 6.3011 mm².
NCT00433017: Verteporfin with Ranibizumab vs. Ranibizumab Alone
Another trial (NCT00433017) evaluated Verteporfin combined with ranibizumab compared to ranibizumab alone. At 12 months:
- Retinal Thickness: Both treatments reduced central retinal thickness. The combination therapy of Verteporfin plus ranibizumab led to a mean reduction of -115.3 µm, slightly more than ranibizumab alone, which reduced thickness by -107.7 µm.
- Treatment-Free Interval: A higher percentage of participants on combination therapy (95.8%) had a treatment-free interval of at least 3 months after the month 2 visit, compared to 92.2% on ranibizumab alone. This suggests potentially less frequent injections with combination therapy.
- Visual Acuity: Ranibizumab alone resulted in a mean gain of 4.4 letters in best-corrected visual acuity, while the combination with Verteporfin resulted in a mean gain of 2.5 letters.
NCT00436553: Verteporfin (Reduced/Standard Fluence) with Ranibizumab
A study (NCT00436553) compared ranibizumab monotherapy with ranibizumab combined with either reduced fluence (RF) or standard fluence (SF) Verteporfin. At 12 months:
- Visual Acuity: Ranibizumab monotherapy showed the greatest mean gain in best-corrected visual acuity, with an average increase of 8.1 letters. The combination with standard fluence Verteporfin resulted in a 5.3-letter gain, and with reduced fluence Verteporfin, a 4.4-letter gain.
- Retinal Thickness: Ranibizumab monotherapy led to the largest mean reduction in central retinal thickness (-172.238 µm), compared to -151.733 µm with standard fluence combination and -140.919 µm with reduced fluence combination.
- Treatment-Free Interval: A treatment-free interval of at least 3 months was achieved by 92.6% of participants in the standard fluence combination group, compared to 83.5% in the reduced fluence combination group.
NCT00473642: Reduced Fluence PDT with Ranibizumab
In a study (NCT00473642) comparing ranibizumab monotherapy to ranibizumab with reduced or standard fluence PDT (photodynamic therapy using Verteporfin):
- Visual Acuity: Ranibizumab monotherapy resulted in the highest mean gain in best-corrected visual acuity, with an average increase of 16.4 letters. Reduced fluence PDT with ranibizumab led to a 7.6-letter gain, and standard fluence PDT with ranibizumab to a 5.9-letter gain.
- Retinal Thickness: Reduced fluence PDT with ranibizumab showed the greatest mean central macular thickness reduction of 76.1 µm, compared to 42.9 µm with standard fluence PDT and 34.9 µm with ranibizumab monotherapy.
- Ranibizumab Retreatments: Both combination therapies required fewer ranibizumab retreatments over 12 months. Standard fluence PDT with ranibizumab required an average of 2.67 retreatments, and reduced fluence PDT with ranibizumab required 2.91 retreatments, compared to 4.14 retreatments for ranibizumab monotherapy.
NCT00492284: Verteporfin with Ranibizumab and Dexamethasone (RADICAL)
The RADICAL study (NCT00492284) explored combinations of reduced fluence Verteporfin, ranibizumab, and dexamethasone. At 12 months:
- Visual Acuity: The 1/2 fluence triple therapy (Verteporfin + ranibizumab + dexamethasone) showed the highest mean gain in best-corrected visual acuity, with an average increase of 6.8 letters. Ranibizumab alone resulted in a 6.5-letter gain.
- Retinal Thickness: All combination therapies resulted in greater mean reductions in central retinal thickness compared to ranibizumab alone. The 1/2 fluence triple therapy achieved a mean reduction of -122.2 µm, and 1/2 fluence double therapy (Verteporfin + ranibizumab) achieved -121.0 µm, compared to -100.3 µm with ranibizumab alone.
- Retreatments: The 1/4 fluence triple therapy and 1/2 fluence triple therapy groups required fewer mean retreatments (3.97 and 4.34, respectively) compared to ranibizumab alone (5.39 retreatments).
Currently Recruiting Trials
Verteporfin is currently being investigated in several clinical trials for a range of conditions, from various cancers to eye disorders. These studies aim to evaluate its safety and effectiveness, often in combination with other therapies or systems.
One ongoing Phase 1/Phase 2 trial, NCT06807359, is exploring photodynamic therapy (PDT) using the SpectraCure P18 System with verteporfin for injection (VFI) to treat primary localized prostate cancer. This study seeks to gather safety data, establish optimal dose parameters, and assess treatment effectiveness, with an enrollment target of 43 participants.
For patients with unresectable, locally advanced, or metastatic pancreatic cancer, a Phase 2 trial, NCT06381154, is testing photoradiation with verteporfin in combination with pembrolizumab and standard chemotherapy. This study aims to enroll 25 individuals to evaluate how well this combined approach works.
Another Phase 1/Phase 2 study, NCT06306638, is investigating interstitial photodynamic therapy (I-PDT) with verteporfin following palliative radiotherapy for inoperable malignant central airway obstruction. This trial, targeting 42 participants, is studying the side effects and efficacy of this treatment for advanced lung cancer tumors that cause breathing difficulties.
In the field of ophthalmology, a study identified as NCT05589974 is monitoring choroidal blood flow in patients with acute and chronic central serous chorioretinopathy (CSC). This study aims to enroll 50 participants to unveil the flow and pulse wave characteristics of the choroidal circulation in CSC.
For recurrent high-grade EGFR-mutated glioblastoma, a Phase 1/Phase 2 trial, NCT04590664, is studying the side effects and best dose of Visudyne (liposomal verteporfin) and its effectiveness. This trial is recruiting 24 participants.
Finally, a Phase 1/Phase 2 clinical study, NCT03067051, is assessing the safety and efficacy of the SpectraCure P18 System, utilizing verteporfin for injection (VFI), for the treatment of recurrent prostate cancer. This study aims to enroll 66 participants to establish dose parameters and gather efficacy data.
Where to Participate
Clinical trials involving verteporfin are conducted across various locations, offering opportunities for participation in different regions. Currently, these studies are active in 5 cities across 4 states in the United States.
The top locations for these recruiting trials include:
- New York, New York (2 sites)
- Atlanta, Georgia (1 site)
- Rochester, Minnesota (1 site)
- Buffalo, New York (1 site)
- Philadelphia, Pennsylvania (1 site)
Eligibility criteria for these trials generally require participants to be between 18 and 70 years of age. All genders are welcome to participate, but these studies are not open to healthy volunteers or children, focusing specifically on patients with the conditions being investigated.
Development Timeline
The clinical development of verteporfin began with the first trial initiated on November 19, 2002, and continues with studies projected to run until at least March 23, 2026. Over this period, a total of 46 clinical trials have been conducted or are ongoing, involving an estimated 3,386 participants.
Early investigations initially explored conditions such as IBS-C and hyperphosphatemia. However, the development pipeline quickly expanded, with a significant focus emerging on various eye conditions. This includes Polypoidal Choroidal Vasculopathy, Chronic Central Serous Chorioretinopathy, Neovascular Age-related Macular Degeneration, and Age-Related Macular Degeneration, among others. More recently, the scope has broadened to include several types of cancer, such as Prostate Cancer, Recurrent Prostate Cancer, Locally Advanced Pancreatic Adenocarcinoma, Metastatic Pancreatic Adenocarcinoma, Glioblastoma, and Malignant Solid Neoplasm.
The trials have progressed through various phases, with a notable presence in Phase 2 (13 trials), Phase 1/Phase 2 (8 trials), and Phase 3 (8 trials), indicating a sustained effort in both early-stage exploration and advanced efficacy testing. Key sponsors driving this research include **Novartis**, **QLT Inc.**, **Mayo Clinic**, and **SpectraCure AB**, alongside numerous other academic institutions and pharmaceutical companies, reflecting a collaborative effort in advancing verteporfin's potential applications.