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RARE DISEASE
Central retinal vein occlusion
Central retinal vein occlusion
Central retinal vein occlusion
Synonyms: CRVO
Synonyms: CRVO
Synonyms: CRVO
Drug discovery
1
drug
With orphan designation
Overview
Central retinal vein occlusion (CRVO) is a vision-threatening retinal vascular disorder characterized by thrombosis of the central retinal vein, leading to venous stasis, retinal hemorrhage, and macular edema [1][4][9]. Predominantly affecting individuals >50 years, key risk factors include hypertension, diabetes, glaucoma, and atherosclerosis [1][14]. Vision loss stems from ischemic retinal damage or macular edema [4][9]. First-line therapy involves intravitreal anti-VEGF agents (aflibercept, ranibizumab) and corticosteroids, supplemented by laser therapy for neovascular complications [3][7][9].
Population
Primarily occurs in adults >50 years (90% of cases), with hypertension present in 73% of patients [14][10]
Younger patients (<40 years) account for 8-15% of cases, often linked to ocular hypertension, inflammation, or coagulopathies [2][6]
Incidence increases with age, slightly male-predominant (53.8%), and 6-17% risk of bilateral involvement [2][14]
Burden
Affects 4.67 million globally (0.13% prevalence), with 30-50% developing permanent vision impairment [6][10]
Annual healthcare costs exceed $1.5 billion (US) due to frequent monitoring/injections [4][6]
Macular edema occurs in 75% of cases; 50% develop neovascular glaucoma without intervention [9][14]
Therapies
Anti-VEGF therapy: First-line for macular edema (≥3 lines VA improvement in 46-58% with monthly injections) [3][7][9]
Steroids: Dexamethasone implants or triamcinolone for anti-VEGF-resistant cases, despite cataract/glaucoma risks [3][7][12]
Laser photocoagulation: Prevents neovascular glaucoma in ischemic CRVO (50% rubeosis risk without treatment) [9][14]
Categories: rare ophthalmic disorders
Research Papers
2,078 drug discovery papers related to Central retinal vein occlusion, with 4 first-in-class and 0 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:
2,078 drug discovery papers related to Central retinal vein occlusion, with 4 first-in-class and 0 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:
2026-07-10 | Effectiveness and safety of intravitreal faricimab for macular oedema secondary to retinal vein occlusion: a systematic review and meta-analysis.
Faricimab is a bispecific monoclonal antibody targeting both vascular endothelial growth factor A and angiopoietin-2, approved for macular oedema secondary to retinal vein occlusion in 2023. The phase III BALATON and COMINO trials demonstrated non-inferiority to aflibercept at week 24, and several real-world cohorts have subsequently emerged. The aim of this systematic review and meta-analysis was to estimate the pooled effect of faricimab on visual acuity, treatment burden, anatomical outcomes, and safety in this indication. PubMed, EMBASE, and the Cochrane Central Register of Controlled Trials were systematically searched from inception to 1 May 2026 for studies reporting outcomes following intravitreal faricimab. Risk of bias was assessed in duplicate using the Cochrane Risk of Bias 2 tool for the randomised evidence and the Risk Of Bias In Non-randomised Studies of Interventions tool for the observational evidence, with single-arm cohorts evaluated as pre-post comparisons. The primary visual acuity outcome was pooled using random-effects meta-analysis with Hartung-Knapp-Sidik-Jonkman adjustment, stratified by treatment status, while outcomes precluded from pooling by methodological heterogeneity were synthesised narratively. Certainty of evidence was assessed using the Grading of Recommendations, Assessment, Development and Evaluation framework. Ten studies comprising 1,620 eyes met eligibility criteria, including the BALATON and COMINO randomised controlled trials and nine non-randomised cohorts. The pooled mean change in best-corrected visual acuity at approximately 6 months was + 16.89 Early Treatment Diabetic Retinopathy Study letters (95% confidence interval 16.05 to 17.72) in treatment-naïve eyes and + 8.73 letters (95% confidence interval 4.75 to 12.71) in refractory switch cohorts, with negligible between-study heterogeneity in both analyses. Narrative synthesis was consistent with treatment interval extension following initiation of or switch to faricimab, statistically significant reductions in central retinal thickness across studies, and a short-term safety profile without identified retinal vasculitis events. The synthesis is consistent with intravitreal faricimab use being associated with visual and anatomical improvement in macular oedema secondary to retinal vein occlusion, with the treatment-naïve pooled estimate primarily reflecting registration trial data and the switch cohort estimate characterising refractory phenotypes.
2026-07-01 | Timing of anti-VEGF therapy and postoperative macular edema after cataract surgery in eyes with retinal vein occlusion: a retrospective cohort study.
Patients with retinal vein occlusion (RVO) undergoing cataract extraction are at increased risk of postoperative macular edema (pME), however, optimal perioperative management strategies remain unclear. This study evaluated the incidence of pME in patients with prior RVO undergoing cataract extraction and assessed the effect of anti-VEGF timing on pME outcomes. A retrospective cohort study at a single institution was conducted by chart review (2013-2023) using ICD-10 codes for RVO and CPT codes for subsequent cataract extraction in the same eye. Exclusion criteria included diabetic macular edema, lack of perioperative optical coherence tomography, and complex cataract extraction. A total of 53 eyes from 51 patients met study criteria. Data collected included demographics, medical history, ocular history, pre- and postoperative central subfield thickness, average cube thickness, timing of anti-VEGF relative to cataract extraction, fluorescein angiography, and postoperative topical medications. pME was defined as a > 30% increase in CST from baseline. Univariate and multivariate logistic regression were performed to identify independent risk factors for pME. The cohort had a mean age of 74.3 ± 9.9 years; 56.6% of eyes had branch RVO and 43.4% had central RVO. The overall incidence of pME was 26.1%, with mean time to development of 48.1 ± 25.1 days. A total of 38 eyes (71.7%) had previously received anti-VEGF therapy. Eyes receiving anti-VEGF within 35 days prior to cataract extraction had a significantly lower incidence of pME (12.5%, n = 24) compared with those treated more than 35 days before surgery (57.1%, n = 14; p = 0.033). Among eyes with ischemic RVO on fluorescein angiography, none receiving anti-VEGF within 35 days developed pME, whereas 80.0% of those treated outside this interval developed pME (p = 0.002). On multivariate logistic regression, pretreatment with anti-VEGF remained the only significant independent factor (adjusted OR 0.041, 95% CI 0.004-0.466, p = 0.010), after adjustment for ischemic status and diabetes. Patients with RVO have a higher risk of developing postoperative macular edema after cataract extraction. Anti-VEGF pretreatment within 35 days before cataract extraction was associated with lower incidence of pME, particularly in cases of ischemic RVO. These findings suggest that perioperative anti-VEGF timing may influence postoperative outcomes, warranting further prospective investigation.
2026-06-25 | The Impact of PCSK9 Inhibitors on Development of Retinal Vascular Occlusions.
PCSK9 inhibitors (PCSK9i) are a newer class of lipid-lowering drug that may be effective at lowering risk for retinal artery (RAO) and retinal vein occlusion (RVO). This study aims to investigate this relationship between PCSK9i use and retinal vascular occlusion among patients with hyperlipidemia. Retrospective, comparative clinical cohort study SUBJECTS, PARTICIPANTS, AND/OR CONTROLS: : Patients with hyperlipidemia, defined as serum LDL level of ≥130 mg/dL and total cholesterol level of ≥220 mg/dL, that were prescribed a lipid-lowering medication were identified. Patients prescribed a PCSK9i were included in the study group and were compared to control patients prescribed any other type of lipid-lowering drug. This study was conducted utilizing electronic health record data from health organizations in the United States through the TrinetX platform. Propensity-score matching (PSM) was completed based on relevant patient demographics, comorbidities, and lab values. Comparison of main outcomes between the PCSK9i and non-PCSK9i groups was performed using measures of association analysis to determine risk ratio (RR) with 95% confidence intervals (CI). The outcomes measured consisted of occurrence of retinal vascular occlusion, RAO, RVO, central RAO, and central RVO at 3-year, 5-year, and 7-year time points. Following PSM, a total of 12,960 patients were included in each cohort. The analysis revealed the PCSK9i cohort had a significantly lower risk for development of retinal vascular occlusions at multiple points, including 3-year (RR 0.56, CI 0.39-0.79), 5-year (RR 0.50, CI 0.37-0.67) and 7-year time points (RR 0.46, CI 0.35-0.61). This lower risk was also seen in the PCSK9i group for an outcome of RVO at 5-years (RR 0.50, CI 0.34-0.73) and 7-years (RR 0.47, CI 0.33-0.67). For occurrence of RAOs (RR 0.47, CI 0.30-0.76) and CRVO (RR 0.46, CI 0.29-0.74) separately, the PCSK9i cohort had a lower risk at 7 years. These findings suggest that PCSK9i may reduce the risk of retinal vascular occlusion compared to other classes of lipid-lowering medications. PRéCIS: Patients prescribed PCSK9 inhibitors had a significantly lower risk of developing retinal vascular occlusions, including venous and arterial occlusions, over seven years compared to those on other lipid-lowering therapies, suggesting protective microvascular effects.
2026-07-10 | Effectiveness and safety of intravitreal faricimab for macular oedema secondary to retinal vein occlusion: a systematic review and meta-analysis.
Faricimab is a bispecific monoclonal antibody targeting both vascular endothelial growth factor A and angiopoietin-2, approved for macular oedema secondary to retinal vein occlusion in 2023. The phase III BALATON and COMINO trials demonstrated non-inferiority to aflibercept at week 24, and several real-world cohorts have subsequently emerged. The aim of this systematic review and meta-analysis was to estimate the pooled effect of faricimab on visual acuity, treatment burden, anatomical outcomes, and safety in this indication. PubMed, EMBASE, and the Cochrane Central Register of Controlled Trials were systematically searched from inception to 1 May 2026 for studies reporting outcomes following intravitreal faricimab. Risk of bias was assessed in duplicate using the Cochrane Risk of Bias 2 tool for the randomised evidence and the Risk Of Bias In Non-randomised Studies of Interventions tool for the observational evidence, with single-arm cohorts evaluated as pre-post comparisons. The primary visual acuity outcome was pooled using random-effects meta-analysis with Hartung-Knapp-Sidik-Jonkman adjustment, stratified by treatment status, while outcomes precluded from pooling by methodological heterogeneity were synthesised narratively. Certainty of evidence was assessed using the Grading of Recommendations, Assessment, Development and Evaluation framework. Ten studies comprising 1,620 eyes met eligibility criteria, including the BALATON and COMINO randomised controlled trials and nine non-randomised cohorts. The pooled mean change in best-corrected visual acuity at approximately 6 months was + 16.89 Early Treatment Diabetic Retinopathy Study letters (95% confidence interval 16.05 to 17.72) in treatment-naïve eyes and + 8.73 letters (95% confidence interval 4.75 to 12.71) in refractory switch cohorts, with negligible between-study heterogeneity in both analyses. Narrative synthesis was consistent with treatment interval extension following initiation of or switch to faricimab, statistically significant reductions in central retinal thickness across studies, and a short-term safety profile without identified retinal vasculitis events. The synthesis is consistent with intravitreal faricimab use being associated with visual and anatomical improvement in macular oedema secondary to retinal vein occlusion, with the treatment-naïve pooled estimate primarily reflecting registration trial data and the switch cohort estimate characterising refractory phenotypes.
2026-07-01 | Timing of anti-VEGF therapy and postoperative macular edema after cataract surgery in eyes with retinal vein occlusion: a retrospective cohort study.
Patients with retinal vein occlusion (RVO) undergoing cataract extraction are at increased risk of postoperative macular edema (pME), however, optimal perioperative management strategies remain unclear. This study evaluated the incidence of pME in patients with prior RVO undergoing cataract extraction and assessed the effect of anti-VEGF timing on pME outcomes. A retrospective cohort study at a single institution was conducted by chart review (2013-2023) using ICD-10 codes for RVO and CPT codes for subsequent cataract extraction in the same eye. Exclusion criteria included diabetic macular edema, lack of perioperative optical coherence tomography, and complex cataract extraction. A total of 53 eyes from 51 patients met study criteria. Data collected included demographics, medical history, ocular history, pre- and postoperative central subfield thickness, average cube thickness, timing of anti-VEGF relative to cataract extraction, fluorescein angiography, and postoperative topical medications. pME was defined as a > 30% increase in CST from baseline. Univariate and multivariate logistic regression were performed to identify independent risk factors for pME. The cohort had a mean age of 74.3 ± 9.9 years; 56.6% of eyes had branch RVO and 43.4% had central RVO. The overall incidence of pME was 26.1%, with mean time to development of 48.1 ± 25.1 days. A total of 38 eyes (71.7%) had previously received anti-VEGF therapy. Eyes receiving anti-VEGF within 35 days prior to cataract extraction had a significantly lower incidence of pME (12.5%, n = 24) compared with those treated more than 35 days before surgery (57.1%, n = 14; p = 0.033). Among eyes with ischemic RVO on fluorescein angiography, none receiving anti-VEGF within 35 days developed pME, whereas 80.0% of those treated outside this interval developed pME (p = 0.002). On multivariate logistic regression, pretreatment with anti-VEGF remained the only significant independent factor (adjusted OR 0.041, 95% CI 0.004-0.466, p = 0.010), after adjustment for ischemic status and diabetes. Patients with RVO have a higher risk of developing postoperative macular edema after cataract extraction. Anti-VEGF pretreatment within 35 days before cataract extraction was associated with lower incidence of pME, particularly in cases of ischemic RVO. These findings suggest that perioperative anti-VEGF timing may influence postoperative outcomes, warranting further prospective investigation.
2026-06-25 | The Impact of PCSK9 Inhibitors on Development of Retinal Vascular Occlusions.
PCSK9 inhibitors (PCSK9i) are a newer class of lipid-lowering drug that may be effective at lowering risk for retinal artery (RAO) and retinal vein occlusion (RVO). This study aims to investigate this relationship between PCSK9i use and retinal vascular occlusion among patients with hyperlipidemia. Retrospective, comparative clinical cohort study SUBJECTS, PARTICIPANTS, AND/OR CONTROLS: : Patients with hyperlipidemia, defined as serum LDL level of ≥130 mg/dL and total cholesterol level of ≥220 mg/dL, that were prescribed a lipid-lowering medication were identified. Patients prescribed a PCSK9i were included in the study group and were compared to control patients prescribed any other type of lipid-lowering drug. This study was conducted utilizing electronic health record data from health organizations in the United States through the TrinetX platform. Propensity-score matching (PSM) was completed based on relevant patient demographics, comorbidities, and lab values. Comparison of main outcomes between the PCSK9i and non-PCSK9i groups was performed using measures of association analysis to determine risk ratio (RR) with 95% confidence intervals (CI). The outcomes measured consisted of occurrence of retinal vascular occlusion, RAO, RVO, central RAO, and central RVO at 3-year, 5-year, and 7-year time points. Following PSM, a total of 12,960 patients were included in each cohort. The analysis revealed the PCSK9i cohort had a significantly lower risk for development of retinal vascular occlusions at multiple points, including 3-year (RR 0.56, CI 0.39-0.79), 5-year (RR 0.50, CI 0.37-0.67) and 7-year time points (RR 0.46, CI 0.35-0.61). This lower risk was also seen in the PCSK9i group for an outcome of RVO at 5-years (RR 0.50, CI 0.34-0.73) and 7-years (RR 0.47, CI 0.33-0.67). For occurrence of RAOs (RR 0.47, CI 0.30-0.76) and CRVO (RR 0.46, CI 0.29-0.74) separately, the PCSK9i cohort had a lower risk at 7 years. These findings suggest that PCSK9i may reduce the risk of retinal vascular occlusion compared to other classes of lipid-lowering medications. PRéCIS: Patients prescribed PCSK9 inhibitors had a significantly lower risk of developing retinal vascular occlusions, including venous and arterial occlusions, over seven years compared to those on other lipid-lowering therapies, suggesting protective microvascular effects.
Access all drug discovery articles and probability of success in trials forecasts:
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Drug Discovery Landscape
1 orphan drug designation for Central retinal vein occlusion.
1 orphan drug designation for Central retinal vein occlusion.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Aganirsen | oligonucleotides | EMA | 2014-06-10 | — | Gene Signal SAS |
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