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Netarsudil

Netarsudil is a topical ophthalmic approved for reducing elevated in patients with open-angle or . Marketed under the brand name Rhopressa as a 0.02% sterile , it represents the first commercially available medication in its class in the United States to target the for enhanced aqueous humor outflow. Netarsudil's primary involves inhibition of rho-associated coiled-coil containing protein kinase (), which relaxes the and to facilitate greater conventional outflow of aqueous humor from the eye, thereby lowering (IOP). It also exhibits () inhibition, contributing to its IOP-lowering effects through modulation of episcleral venous pressure. The U.S. (FDA) approved netarsudil on December 18, 2017, with subsequent approvals in (as Rhokiinsa) in 2019 and other regions including as of 2025, based on phase 3 clinical trials (ROCKET 1 and 2) demonstrating mean IOP reductions of approximately 4–5 mmHg when used as monotherapy, comparable to timolol but with a distinct mechanism addressing the primary site of outflow resistance in . Clinically, netarsudil is administered as one drop in the affected eye(s) once daily, preferably in the evening, and can be used alone or adjunctively with other ocular hypotensive agents, with a 5-minute interval between instillations. Efficacy data from randomized controlled trials indicate sustained IOP lowering over 12 months, with additional benefits in combination formulations like netarsudil/latanoprost (Rocklatan), which showed superior hypotensive effects compared to either component alone in meta-analyses. Systemic exposure is minimal due to rapid ocular metabolism by esterases to its active form, netarsudil-M1, with no detectable concentrations in most patients. The most common adverse reactions include conjunctival hyperemia (affecting up to 53% of patients), corneal verticillata (whorl-like deposits, typically asymptomatic and reversible), and instillation site pain (15–20%), with overall ocular tolerability supported by low discontinuation rates in long-term studies. No significant systemic concerns have been identified, though suggests slightly attenuated IOP reduction in diverse populations compared to controlled trials. Ongoing research explores its potential in pediatric and corneal endothelial disorders, highlighting its versatility beyond traditional glaucoma management.

Medical uses

Indications

Netarsudil, marketed as Rhopressa, is a topical ophthalmic solution indicated for the reduction of elevated (IOP) in adult patients with open-angle or . This approval, granted by the U.S. in December 2017, targets the primary open-angle form of , which is the most common subtype, as well as cases of elevated IOP without glaucomatous . The medication addresses these conditions by enhancing aqueous humor outflow through the , thereby lowering IOP to prevent damage and vision loss associated with progression. Safety and efficacy of netarsudil have not been established in pediatric patients under 18 years of age, limiting its approved use to adults. In elderly patients, no overall differences in safety or effectiveness were observed compared to younger adults, though individual responses may vary due to age-related factors such as concurrent medical conditions or . Netarsudil may be prescribed as monotherapy for initial treatment or as adjunctive therapy in combination with other IOP-lowering agents, such as beta-blockers or analogs, to achieve additive IOP reduction in cases.

Administration

Netarsudil is administered as a 0.02% ophthalmic solution in the form of preservative-containing , typically dispensed in 2.5 mL multi-dose bottles. The recommended dosage is one drop instilled into the affected eye or eyes once daily, preferably in the evening, to maintain consistent reduction. If a dose is missed, the next dose should be administered as scheduled without doubling up, as twice-daily dosing is not recommended due to potential increased risk of side effects. To administer the drops, patients should begin by washing their hands thoroughly with and to minimize . The bottle should be shaken gently if needed, and the dropper tip inspected for any damage or foreign particles. Tilt the head back slightly and use a finger to pull down the lower , creating a small pocket. Hold the dropper tip close to the eye without touching the eye, , or surrounding areas, then squeeze the bottle to release one drop into the pocket. After instillation, close the eye gently for 2 to 3 minutes while looking downward, and apply light pressure to the inner corner of the eye (near the ) to prevent into the tear duct. Avoid or rubbing the eye immediately after. Any excess solution around the eye can be wiped away with a clean tissue, the cap replaced tightly, and hands washed again. When using netarsudil with other topical ocular medications, each should be administered at least 5 minutes apart to ensure proper absorption and avoid dilution. Contact lenses must be removed prior to administration because the preservative can be absorbed by soft lenses, potentially causing discoloration or irritation; lenses may be reinserted 15 minutes after the drops are instilled. To prevent bacterial contamination and the risk of serious eye infections like , the dropper tip must never contact the eye or any surface, and the bottle should not be shared.

Safety profile

Contraindications

Netarsudil ophthalmic solution, marketed as Rhopressa , has no specific contraindications listed in the FDA-approved prescribing information. In the , where it is marketed as Rhokiinsa, the only is hypersensitivity to the active substance (netarsudil mesylate) or to any of the excipients. As is standard for all topical ophthalmic medications, netarsudil should not be used in patients with a known or suspected to netarsudil or any component of the formulation, as this may result in allergic reactions such as , swelling, or difficulty breathing. No contraindications related to , , renal or hepatic impairment, or concurrent ocular conditions are specified in regulatory labeling, though clinical judgment is recommended in such cases due to limited data.

Adverse effects

Netarsudil, administered as an ophthalmic solution, is generally well-tolerated, with adverse effects primarily affecting the ocular surface and occurring in a significant proportion of patients during clinical use. The most frequently reported adverse reaction is conjunctival hyperemia, observed in 53% of patients in phase 3 trials, often mild to moderate in severity and tending to decrease over the first month of treatment. Other common ocular adverse effects, occurring in approximately 20% of patients, include corneal verticillata (whorl-like epithelial deposits), instillation site pain, and conjunctival hemorrhage. Corneal verticillata typically appears within 4 weeks of initiation, does not impair visual function, and resolves upon discontinuation without long-term sequelae. Conjunctival hemorrhage is usually self-limiting and resolves with continued use. Less common effects (5-10% incidence) encompass instillation site , corneal staining, , increased lacrimation, eyelid , and reduced . Postmarketing experience has reported epithelial corneal associated with netarsudil use; patients should be monitored for corneal changes. In real-world settings, adverse effects are reported in about 40% of patients, with conjunctival hyperemia remaining the most prevalent at around 30%, and discontinuation due to intolerance occurring in 10-12% of cases. Rare but notable effects include punctal stenosis, which may require discontinuation, and long-term use has been associated with crystalline keratopathy in isolated cases, potentially leading to persistent corneal deposits and impairment, particularly in patients with preexisting ocular surface . A 2025 pharmacovigilance analysis of the FDA Reporting System (FAERS) database identified 63 adverse event signals, mostly confirming known ocular effects like conjunctival hyperemia and , but also new signals including allergic , eye pruritus, dacryostenosis, myopic shift, corneal hemorrhage, and rare systemic reactions such as facial swelling and allergic . Systemic adverse effects are uncommon due to low absorption, with mild headaches reported infrequently. Patients should be monitored for signs of bacterial , especially those with corneal abnormalities, as multi-dose containers pose a risk. Contact lens wearers must remove lenses prior to instillation and wait 15 minutes before reinsertion. Overall, most adverse effects are transient and localized, supporting netarsudil's favorable safety profile for management.

Overdose

Netarsudil is administered as a topical ophthalmic with minimal systemic absorption, making clinically significant overdose unlikely when used as directed. No specific on overdose symptoms or outcomes are available in the prescribing information or clinical studies, as systemic exposure remains low even with standard dosing. In cases of suspected overdose, such as accidental of the or excessive ocular application, immediate medical evaluation is recommended. Contact a (e.g., 1-800-222-1222 in the United States) or seek emergency care, providing details on the amount ingested and timing. Supportive care should be administered based on symptoms, though no targeted exists. Preclinical studies in animals indicate potential toxicity at high intravenous doses, including embryofetal effects in rabbits and rats, but these findings are not directly applicable to human topical use due to the route of administration and low bioavailability.

Interactions

With other ocular medications

Netarsudil, a used to reduce (IOP) in patients with open-angle or , exhibits no significant drug-drug interactions with other topical ophthalmic medications. This lack of interactions allows for safe concomitant use with various ocular hypotensive agents, such as analogs, beta-blockers, and inhibitors, to achieve additive IOP-lowering effects. When administered alongside other topical , netarsudil should be spaced at least 5 minutes apart to minimize potential dilution or washout effects and ensure optimal absorption of each medication. Clinical studies have demonstrated its compatibility in ; for instance, netarsudil added to latanoprost monotherapy resulted in an additional mean IOP reduction of approximately 1.4–2.6 mmHg, without reported adverse pharmacokinetic or safety interactions. Similarly, use with timolol has shown additive benefits, with netarsudil providing further IOP lowering of about 0.9–1.6 mmHg compared to monotherapy. Real-world evidence supports the tolerability of netarsudil in polytherapy regimens, where it is often added to existing treatments involving up to four other ocular hypotensives, yielding modest additional IOP reductions (around 2–3 mmHg) while maintaining a favorable safety profile. No contraindications or enhanced adverse effects have been identified in these combinations, though routine monitoring for common side effects like conjunctival hyperemia is recommended.

Administration considerations

Netarsudil ophthalmic solution is administered as one drop instilled into the affected eye(s) once daily, preferably in the evening, to reduce elevated in patients with open-angle or . If a dose is missed, patients should continue with the next scheduled dose without doubling up. Twice-daily dosing is not recommended due to potential increased risk of adverse effects without additional efficacy. Proper administration technique is essential to maximize efficacy and minimize risks. Patients should wash their hands thoroughly before use, avoid touching the dropper tip to the eye or any surface to prevent contamination and bacterial keratitis, tilt their head back, pull down the lower to form a , instill without touching the eye, close the eye for 2-3 minutes, apply gentle pressure to the inner corner of the eye (nasolacrimal ) for at least one minute to reduce systemic absorption, and wipe away any excess solution. When using netarsudil with other topical ophthalmic medications, each product should be administered at least 5 minutes apart to ensure optimal absorption and avoid dilution. Contact lens wearers must remove lenses prior to instillation of netarsudil and wait at least 15 minutes before reinserting them, as the solution contains benzalkonium chloride, a that may be absorbed by soft . The product is supplied in a sterile, multi-dose and should be stored refrigerated (2°C to 8°C or 36°F to 46°F) until opened; after opening, it may be kept at (up to 25°C or 77°F) for up to 6 weeks or until the expiration date, whichever comes first. Patients with corneal disease or a history of epithelial defects are at higher risk for contamination-related complications and require careful monitoring. Healthcare providers should counsel patients on these procedures to promote adherence and safety, including reporting any signs of or promptly. Regular intraocular pressure monitoring is advised following initiation to assess response, as the maximum IOP-lowering effect may take up to 7 days to achieve.

Pharmacology

Mechanism of action

Netarsudil is a dual-action ophthalmic agent that functions primarily as an inhibitor of Rho-associated coiled-coil containing (ROCK), specifically targeting ROCK1 and ROCK2 isoforms, which are serine/ kinases downstream of Rho signaling. By inhibiting ROCK, netarsudil reduces actomyosin contraction in the (TM) and Schlemm's canal (SC) cells, thereby decreasing cellular stiffness, extracellular matrix deposition, and expression of fibrosis-related proteins. This relaxation of the TM enhances aqueous humor outflow through the conventional trabecular pathway, increasing outflow facility by promoting TM tissue expansion, enlarging the effective filtration area, and dilating episcleral veins. Additionally, netarsudil inhibits the norepinephrine transporter (NET), preventing the reuptake of norepinephrine and thereby potentiating its signaling effects. This leads to vasoconstriction in the ciliary body vasculature, which reduces aqueous humor production at the ciliary epithelium. The combined effects of increased trabecular outflow and decreased aqueous humor production, along with a reduction in episcleral venous pressure, contribute to netarsudil's intraocular pressure (IOP)-lowering efficacy in conditions such as open-angle glaucoma and ocular hypertension. Preclinical studies in animal models have demonstrated these mechanisms, with netarsudil increasing outflow facility by up to 53% in normotensive monkey eyes and significantly lowering episcleral venous pressure in rabbits. Upon topical administration, netarsudil is rapidly metabolized in the by esterases to its more potent , netarsudil-M1, which exhibits approximately five times greater ROCK inhibitory activity and contributes substantially to the therapeutic effect. This dual inhibition distinguishes netarsudil from other IOP-lowering agents that primarily target the uveoscleral pathway, allowing it to address dysfunction in the diseased TM directly.

Pharmacokinetics

Netarsudil, administered as a 0.02% ophthalmic once daily, exhibits low systemic absorption following topical ocular application. In clinical studies involving healthy subjects receiving bilateral doses for 8 days, concentrations of netarsudil were below the lower limit of quantitation (0.100 ng/mL) on both day 1 and day 8, with only one subject showing a quantifiable level of 0.11 ng/mL for the netarsudil-M1 (AR-13503) at 8 hours post-dose on day 8. This minimal systemic exposure underscores its localized action in the eye, minimizing potential off-target effects. Ocular absorption occurs rapidly through the , where netarsudil achieves high concentrations shortly after dosing. Preclinical studies in rabbits demonstrated peak corneal levels of 3,666–3,972 ng·eq/g within 0.25–0.5 hours post-dose, with subsequent penetration into the (AH) reaching Cmax values of 16–23 ng·eq/mL at approximately 8 hours. The drug distributes preferentially to anterior ocular tissues, with exposure highest in the and , followed by the iris-ciliary body, retina-choroid, , vitreous humor, and ; systemic tissues like , liver, and show 200–3,000-fold lower levels compared to ocular sites. Netarsudil undergoes primarily by esterases in the and other ocular tissues, converting it to the netarsudil-M1, which retains pharmacological activity. This occurs across species, including humans, with an of 175 minutes in human corneal tissue. No further metabolism of netarsudil-M1 has been detected, and the process is localized to the eye. Elimination from ocular compartments is relatively slow, supporting once-daily dosing. In models, the in the is 16–17 hours, while elimination from the retina-choroid, , and iris-ciliary body ranges from 68–112 hours. Systemic clearance is negligible due to the lack of measurable levels, with pathways uncharacterized in humans but presumed to involve ocular drainage and minimal hepatic or renal involvement.

Development

Preclinical development

Netarsudil, also known as AR-13324, was discovered through a program initiated in 2006 by Aerie Pharmaceuticals aimed at developing potent inhibitors of Rho-associated coiled-coil containing (ROCK) with dual activity against the (NET) to enhance (IOP) reduction for treatment. The compound emerged from optimization of amino-isoquinoline amide scaffolds, selected for its high potency in biochemical assays (ROCK1/ROCK2 Ki = 1 nM) and its netarsudil-M1 (Ki = 0.2 nM), alongside NET inhibition (Ki = 3 nM). In cell-based assays, netarsudil demonstrated robust effects on trabecular meshwork (TM) cells, reducing actin stress fibers in primary TM cells (IC50 = 79 nM) and focal adhesions in human TM cells (IC50 = 16 nM), mechanisms that promote aqueous humor outflow. It also inhibited transforming growth factor-β2-induced fibrotic markers in human TM cells at concentrations as low as 500 nM, indicating anti-fibrotic potential without cytotoxicity up to 10 μM. These findings supported its role in relaxing TM contractility and remodeling the extracellular matrix, key barriers in glaucoma pathophysiology. Preclinical efficacy was evaluated using Dutch Belted rabbits and Formosan Rock monkeys, models of . Topical administration of 0.04% netarsudil once daily for three days produced dose-dependent IOP reductions, with maximum decreases of 8.1 ± 0.7 mmHg in rabbits and 7.5 ± 1.1 mmHg in monkeys (both P < 0.05 vs. vehicle), sustained for at least 24 hours post-dose. Effects were primarily attributed to enhanced trabecular outflow, with minimal impact on uveoscleral pathways, and were comparable to or exceeded those of prostaglandins like latanoprost in magnitude and duration. Pharmacokinetic studies across species (rabbits, monkeys, rats, dogs) revealed favorable ocular absorption, with high corneal penetration (Cmax 3,666–3,972 ng·eq/g) and low systemic exposure (Cmax < 1 ng/mL). Netarsudil is rapidly metabolized by corneal esterases to netarsudil-M1, the predominant form in aqueous humor (half-life 16–17 hours), with slow elimination from retinal and lens tissues (elimination half-life 68–112 hours). Ocular bioavailability was approximately 50%, supporting once-daily dosing. Safety assessments in preclinical models showed netarsudil to be well-tolerated, with the primary adverse effect being transient, mild conjunctival hyperemia (scoring +0.5 to +1 on a 0–4 scale), resolving within hours and not progressing with repeated dosing. No significant ocular or systemic toxicities were observed in repeat-dose toxicology studies up to 0.04% in rabbits and monkeys over four weeks, nor in genotoxicity, phototoxicity, or cardiovascular safety evaluations. These profiles, combined with potent IOP-lowering efficacy, justified advancement to Phase 1 clinical trials in 2010.

Clinical trials and approvals

Netarsudil ophthalmic solution 0.02% underwent evaluation in multiple phase 3 clinical trials to assess its efficacy and safety for reducing elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. The pivotal ROCKET-1 and ROCKET-2 trials were large, randomized, double-masked studies involving a total of 1,167 patients across both trials, comparing once-daily netarsudil to twice-daily timolol 0.5% after a washout period. In these trials, netarsudil demonstrated noninferiority to timolol, with mean IOP reductions of approximately 6-8 mmHg across time points from 9 AM to 5 PM over 12 months, and it was well tolerated with primarily mild ocular adverse effects. A third phase 3 trial, ROCKET-4, enrolled approximately 700 patients and further confirmed netarsudil's efficacy, showing statistically significant IOP lowering that was noninferior to timolol at primary endpoints (8 AM, 10 AM, and 4 PM measurements over 3 months), with sustained effects through 12 months in open-label extension. Pooled analyses from the ROCKET trials reinforced these findings, reporting consistent IOP reductions of 4.7-7.7 mmHg and a favorable safety profile, with the most common adverse events being conjunctival hyperemia (50-60% incidence, mostly trace to mild) and minimal systemic effects. These trials supported netarsudil's novel mechanism as a Rho-associated protein kinase (ROCK) inhibitor, distinguishing it from traditional IOP-lowering agents. The U.S. Food and Drug Administration (FDA) approved netarsudil ophthalmic solution 0.02% (branded as Rhopressa) on December 18, 2017, for the reduction of elevated IOP in patients with open-angle glaucoma or ocular hypertension, based on the ROCKET trial data. In Europe, the European Medicines Agency (EMA) granted marketing authorization for netarsudil 0.02% eye drops (branded as Rhokiinsa) on November 19, 2019, for the same indications in adults, following review of similar phase 3 evidence. The marketing authorization for Rhokiinsa was transferred to Santen in 2022 to expand commercialization in Europe. Post-approval studies have included trials exploring netarsudil's role in corneal endothelial restoration and prevention of progression after trabeculectomy, but these are investigational and not part of initial approval dossiers. In Japan, a phase 3 trial (J-ROCKET) completed in 2023 compared netarsudil once daily to ripasudil twice daily, demonstrating superior IOP reduction (mean 6.2 mmHg vs. 5.1 mmHg at month 3) and similar tolerability, supporting a regulatory filing submitted in July 2025. Licensing agreements with Santen extend potential approvals to regions including China and India, though specific timelines remain pending as of late 2025.

Society and culture

Brand names

Netarsudil is marketed under the brand name Rhopressa in the United States by Aerie Pharmaceuticals (a subsidiary of Alcon), where it was approved by the Food and Drug Administration on December 18, 2017, for the treatment of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. In the European Union, the drug is sold as Rhokiinsa by Santen Pharmaceutical, following approval by the European Commission on November 19, 2019, and is indicated for the reduction of elevated intraocular pressure in adults with open-angle glaucoma or ocular hypertension. It is also available as Rhopressa in South Korea, launched in November 2024 by Santen Pharmaceutical. As of November 2025, no other primary brand names for monotherapy formulations have been reported in additional markets, though approval applications are pending (e.g., Japan).

Fixed-dose combinations

Netarsudil is available in a fixed-dose combination with latanoprost, a prostaglandin analog, marketed as Rocklatan in the United States (approved by the FDA on March 12, 2019) and as Roclanda in the European Union (approved on January 7, 2021), for the treatment of elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. This combination contains netarsudil 0.02% and latanoprost 0.005% in a single ophthalmic solution, allowing for once-daily administration to simplify treatment regimens and potentially improve patient adherence. In the MERCURY-1 and MERCURY-2 trials, once-daily dosing of the combination achieved statistically and clinically superior IOP reductions compared to monotherapy with either or latanoprost alone, with mean IOP decreases of up to 9.4 mmHg at month 3 across multiple time points. The combination leverages complementary mechanisms: inhibits Rho kinase to enhance trabecular meshwork outflow, while latanoprost increases uveoscleral outflow, resulting in additive IOP-lowering effects without significant increases in adverse events beyond those seen with the individual components. Common ocular side effects include conjunctival hyperemia and corneal verticillata, with discontinuation rates similar to monotherapy arms in long-term studies up to 12 months. Real-world evidence supports its use as an adjunctive therapy; for instance, switching from latanoprost monotherapy to the combination has shown additional IOP reductions of approximately 2-3 mmHg in patients with suboptimal control, maintaining this benefit over 6 months. The combination was launched in Singapore as in March 2025 by Santen Pharmaceutical. No other fixed-dose combinations involving netarsudil have received regulatory approval as of November 2025.

Chemistry

Chemical structure

Netarsudil is an organic compound classified as an amino-isoquinoline amide, featuring a chiral center with (S) configuration. Its molecular formula is C_{28}H_{27}N_{3}O_{3}, and it has a molar mass of 453.54 g/mol. The systematic IUPAC name for netarsudil (free base) is {4-[(1S)-2-amino-1-[(isoquinolin-6-yl)carbamoyl]ethyl]phenyl}methyl 2,4-dimethylbenzoate. This structure comprises a phenyl ring substituted at the para position with a chiral 1-carboxamide-2-aminomethyl ethyl chain, where the carboxamide is linked to the 6-amino group of isoquinoline, and the phenyl ring also bears a hydroxymethyl group esterified with 2,4-dimethylbenzoic acid. The core scaffold includes aromatic (benzene and isoquinoline) and heterocyclic elements, along with amide and ester functional groups that contribute to its pharmacological properties. The canonical SMILES notation is CC1=CC(C)=C(C=C1)C(=O)OCC1=CC=C(C=C1)C@@HC(=O)NC1=CC=C2C=NC=CC2=C1, and the InChI is InChI=1S/C28H27N3O3/c1-18-3-10-25(19(2)13-18)28(33)34-17-20-4-6-21(7-5-20)26(15-29)27(32)31-24-9-8-23-16-30-12-11-22(23)14-24/h3-14,16,26H,15,17,29H2,1-2H3,(H,31,32)/t26-/m1/s1. The compound is typically used clinically as the dimesylate salt, which has the formula C_{30}H_{35}N_{3}O_{9}S_{2} and CAS number 1422144-42-0, enhancing its solubility for ophthalmic formulation.

Physicochemical properties

Netarsudil dimesylate, the active pharmaceutical ingredient in the marketed ophthalmic formulation, is the dimesylate salt of the free base netarsudil. The free base has the molecular formula C28H27N3O3 and a molecular weight of 453.54 g/mol, whereas the dimesylate salt corresponds to C30H35N3O9S2 with a molecular weight of 645.74 g/mol. The compound presents as a light yellow-to-white powder and is characterized as a weak . Its solubility profile supports its formulation as an : it is freely soluble in , soluble in , sparingly soluble in , and practically insoluble in and . In the commercial ophthalmic solution (0.02% w/v (0.2 mg/mL) netarsudil, equivalent to 0.28 mg/mL netarsudil dimesylate), the drug is formulated as a sterile, , buffered with a of approximately 5.0 to 5.7 and an osmolality of about 295 mOsmol/kg. This range aligns with the weakly acidic nature of the dimesylate , facilitating ocular tolerability while maintaining . Predicted physicochemical parameters, such as a value of approximately 3.8 to 4.7 (indicating moderate ) and values of 13.5 (acidic) and 8.7 (basic), further inform its behavior in biological systems, though experimental confirmation in primary literature remains limited.