Verteporfin

Summary

Verteporfin (CHEMBL5314346) is an approved small molecule (ATC S01LA01); indicated across 19 conditions including ocular vascular disorder and age-related macular degeneration; with CIViC clinical evidence for 2 variant-indication associations (e.g. YAP1 Overexpression in esophageal cancer).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: S01LA01
• Indications: 19 conditions
• Clinical trials: 39
• Precision-oncology evidence (CIViC): 2 variant–indication associations

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5314346
Name	Verteporfin
Type	Small molecule
Max phase	4
ATC	S01LA01

Also known as: Bpd-ma, CL 318,952, CL-318952, Verteporfin, Verteporfina, Verteporfine, Visudyne, VERTEPORFIN, verteporfin, NA

Targets

Targets

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Tumour suppressor protein p53/Mdm4, Protein odd-skipped-related 1.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
OSR1	6.7	IC50	200	nM	CHEMBL_ACT_24996396
MDM4	5.95	IC50	1122	nM	CHEMBL_ACT_25872940

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

19 indications (6 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
ocular vascular disorder	4	MONDO:0005552	EFO:0005753
age-related macular degeneration	4	MONDO:0005150	EFO:0001365
degenerative myopia	4	MONDO:0001383	EFO:0004207
macular degeneration	4	MONDO:0003004	EFO:0009606
wet macular degeneration	4	MONDO:0005417	EFO:0004683
basal cell carcinoma	3	MONDO:0020804	EFO:0004193
nevoid basal cell carcinoma syndrome	3	MONDO:0007187	MONDO:0007187
choroidal neovascularization	3	MONDO:0810000	MONDO:0810000
histoplasmosis	2	MONDO:0018312	EFO:0007310
exocrine pancreatic carcinoma	2	MONDO:0005192	EFO:0002618
breast neoplasm	2	MONDO:0021100	MONDO:0007254
pancreatic neoplasm	2	MONDO:0021040	EFO:0003860
cutaneous melanoma	1	MONDO:0005012	EFO:0000389
central nervous system neoplasm	1	MONDO:0006130	EFO:1000158
glioblastoma	1	MONDO:0018177	EFO:0000519
neoplasm	1	MONDO:0005070	EFO:0000616

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 39.

Phase distribution

Phase	Trials
PHASE2	13
PHASE1/PHASE2	8
Not specified	6
PHASE3	5
PHASE4	4
PHASE1	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00135837	PHASE4	COMPLETED	Photodynamic Therapy in Occult-Only Lesions (POOL)
NCT00473642	PHASE4	COMPLETED	Reduced Fluence Photodynamic Therapy (PDT) With Visudyne in Combination With Lucentis for Age-Related Macular Degeneration
NCT02120950	PHASE4	COMPLETED	Aflibercept in Polypoidal Choroidal Vasculopathy
NCT04075136	PHASE4	WITHDRAWN	Optical Coherence Tomography Angiography (OCTA) - Directed PDT Triple Therapy
NCT00121407	PHASE3	COMPLETED	Visudyne® in Occult (VIO)
NCT00370539	PHASE3	UNKNOWN	Combined PDT and Intravitreal Bevacizumab vs Combination of PDT, Intravitreal Bevacizumab and Triamcinolone for Neovascular AMD
NCT00436553	PHASE3	COMPLETED	Efficacy/Safety of Verteporfin Photodynamic Therapy and Ranibizumab Compared With Ranibizumab in Patients With Subfoveal Choroidal Neovascularization
NCT01482910	PHASE3	COMPLETED	VEGF Trap-Eye: Investigation of Efficacy and Safety in Chinese Subjects With Wet AMD (Age-Related Macular Degeneration)
NCT01570608	PHASE3	COMPLETED	Lucentis KAV Study
NCT03067051	PHASE1/PHASE2	RECRUITING	Clinical Study to Assess the Safety and Efficacy of the SpectraCure P18 System
NCT04590664	PHASE1/PHASE2	RECRUITING	Verteporfin for the Treatment of Recurrent High Grade EGFR-Mutated Glioblastoma
NCT06306638	PHASE1/PHASE2	RECRUITING	Interstitial Photodynamic Therapy Following Palliative Radiotherapy in Treating Patients With Inoperable Malignant Central Airway Obstruction
NCT06381154	PHASE2	RECRUITING	Photoradiation With Verteporfin to Facilitate Immunologic Activity of Pembrolizumab in Unresectable, Locally Advanced or Metastatic Pancreatic Cancer
NCT06807359	PHASE1/PHASE2	RECRUITING	Photodynamic Therapy of Primary Localized Prostate Cancer With the SpectraCure P18 System
NCT06944249	PHASE1/PHASE2	NOT_YET_RECRUITING	Effect of YAP1-inhibition in Surgical Wounds.
NCT07488988	PHASE2	NOT_YET_RECRUITING	SCARFREE-001: Verteporfin for Scar Prevention
NCT00007969	PHASE1/PHASE2	COMPLETED	Photodynamic Therapy in Treating Patients With Stage III or Stage IV Melanoma
NCT00140803	PHASE2	COMPLETED	Study Of Combined VISUDYNE Therapy With Kenalog In CNV Secondary To Age-Related Macular Degeneration
NCT00211445	PHASE2	COMPLETED	Photodynamic Therapy Using Verteporfin for Treatment of Chronic Central Serous Chorioretinopathy (CSC)
NCT00390208	PHASE2	COMPLETED	Triple Therapy - PDT Plus IVD and Intravitreal Ranibizumab Versus Lucentis Monotherapy to Treat Age-Related Macular Degeneration
NCT00426998	PHASE2	COMPLETED	Combination Bevacizumab and Verteporfin (Two Different Sequences of Treatment)in Neovascular AMD
NCT00457678	PHASE2	WITHDRAWN	Second Line Therapy to Treat Age-related Macular Degeneration (AMD) for Patients Not Responding Well to Lucentis Therapy by Itself
NCT00492284	PHASE2	COMPLETED	Reduced Fluence Visudyne-Anti-VEGF-Dexamethasone In Combination for AMD Lesions (RADICAL)
NCT00527475	PHASE2	COMPLETED	Ranibizumab and Reduced Fluence PDT for AMD
NCT00546936	PHASE2	UNKNOWN	Ranibizumab Versus PDT for Presumed Ocular Histoplasmosis
NCT00570193	PHASE1/PHASE2	COMPLETED	Photodynamic and Pharmacologic Treatment of CNV
NCT00574093	PHASE2	COMPLETED	Efficacy and Safety Study of Lucentis (Ranibizumab) and Visudyne (Verteporfin) Combination Therapy in Neovascular AMD
NCT01325181	PHASE1/PHASE2	COMPLETED	Ranibizumab Versus Low-fluence Photodynamic Therapy in the Treatment of Chronic Central Serous Chorioretinopathy
NCT02939274	PHASE2	UNKNOWN	An Open Label, Phase II Trial of Continuous Low-Irradiance Photodynamic Therapy (CLIPT) Using Verteporfin (Visudyne®) for the Treatment of Cutaneous Metastases of Breast Cancer
NCT03033225	PHASE2	COMPLETED	Ultrasound-Guided Verteporfin Photodynamic Therapy for the Treatment of Unresectable Solid Pancreatic Tumors or Advanced Pancreatic Cancer, VERTPAC-02 Study
NCT00002647	PHASE1	UNKNOWN	Photodynamic Therapy With Porfimer Sodium in Treating Patients With Refractory Brain Tumors
NCT02464761	PHASE1	COMPLETED	Photodynamic Therapy for the Treatment of Vertebral Metastases
NCT02702700	PHASE1	TERMINATED	Photo-induction as a Means to Improve Cisplatin Delivery to Pleural Malignancies
NCT05589974	Not specified	RECRUITING	Choroidal Blood Flow in Acute and Chronic Central Serous Chorioretinopathy
NCT01746875	Not specified	TERMINATED	A Study Testing if Medicine Can Make Pigment Epithelium Detachments Regress and Stabilize the Vision in Eyes
NCT01974622	Not specified	UNKNOWN	ICG-Guided Photodynamic Therapy for Treatment of Retinal Capillary Abnormalities: A Pilot Study
NCT02457026	Not specified	WITHDRAWN	Adjunctive Photodynamic Therapy + Aflibercept vs. Afilbercept Alone for PDA in NV AMD
NCT02872064	Not specified	COMPLETED	Treatment of Primary Breast Cancer Using PDT
NCT03459144	Not specified	COMPLETED	Comparison of PDT, Intravitreal Ranibizumab and Combination for Polypoidal Choroidal Vasculopathy Under 1+PRN Regimen

Clinical evidence (CIViC)

Variant × indication × effect (2 predictive associations from 2 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
YAP1 Overexpression	Esophageal Cancer	Sensitivity/Response	Verteporfin	CIViC D	EID1983
YAP1 Overexpression	Esophageal Cancer	Sensitivity/Response	Docetaxel + Verteporfin	CIViC D	EID9329

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).