Abstract
In this review period, I am struck by the advanced development and, in some cases, approval of products for ophthalmic diseases that, a generation ago, had no therapy. These include treatments for inherited retinal disease, thyroid eye disease, keratoconus, presbyopia, and myopia. Devices recently listed with FDA include an objective visual field diagnostic machine, as well as treatments for dry eye disease and dry age-related macular degeneration (AMD). In other areas, there is an activity in ophthalmic drug delivery systems, gene and cell therapy, as well as novel molecules being evaluated for the treatment of glaucoma and ocular inflammation. The United States (US) FDA continues to work on many aspects of drug regulation. On the one hand, in calendar 2024, 65 new drugs and biologics were approved, although there were less than a handful of ophthalmical approvals. Meanwhile, the FDA has an important enforcement role, protecting American health by removing unapproved and incorrectly labelled drugs from consumer channels and quality issues with compounded products. The development of targeted biologics a generation ago and the expiration of the proprietary protection has led to biosimilars, including some in ophthalmology. Innovator companies, however, had legal issues with some of these approvals. The availability of novel therapies has a major positive impact on human health—however, at a price. Public policy continues to be concerned with financial impact, including the role of pharmacy benefit managers.
Ophthalmical Pharmaceuticals and Biologics
Aldeyra announced the US FDA has accepted for review its resubmitted new drug application (NDA) for its reproxalap for the treatment of dry eye disease (November 2024).
Alkeus announced interim data from its SAGA and TEASE-3 studies of oral gildeuretinol acetate for patients with Geographic Atrophy and Stargardt’s disease, respectively. The product has also been granted Rare Pediatric Disease and Fast Track designations by the US FDA for the treatment of Stargardt disease (October and November 2024, January 2025).
Cambium Bio received US FDA fast track designation for its CAM-101 (Topical Fibrinogen Depleted Human Platelet Lysate) for the treatment of dry eye disease (December 2024).
Clearside Biomedical announced results from its phase 2b ODYSSEY trial of CLS-AX (axitinib injectable suspension) for the treatment of wet AMD (October 2024).
D. Western Therapeutics Institute announced results from its double-masked, controlled Phase 2B clinical trial of its H-1337 Ophthalmic Solution for the treatment of elevated intraocular pressure in patients with primary open-angle glaucoma or ocular hypertension (November 2024).
Eyenovia presented results from its CHAPERONE study of atropine solution (0.01% and 0.1%) in a proprietary delivery system with a small drop volume (November 2024).
EyePoint Pharmaceuticals reported results from its ongoing phase 2 VERONA study and the commencement of dosing in its 2 phase 3 studies (LUGANO and LUCIA) of Duravyu (EYE-1901 intravitreal delivery system, vorolanib) in the patients with wet AMD (October and December 2024).
Kiora received clearance in Australia to conduct its Phase 2 ABACUS-2 clinical trial for its photoswitch molecule to treat patients with retinitis pigmentosa (RP, October 2024).
MediPrint announced results of its Phase 2b clinical trial of LL-BMT1 (contact lens printed with bimatoprost) in patients with elevated intraocular pressure (November 2024).
Melt Pharmaceuticals presented results for its Phase 3 study of MELT-300 (sublingual midazolam and ketamine) for procedural sedation in patients undergoing cataract surgery (November 2024).
Oculis updated the enrollment status of its phase 3 DIAMOND trials of its OCS-01 (dexamethasone) eye drops in the treatment of diabetic macular edema (DME; October 2024). The firm also reported Phase 2 data on its OCS-05 intravenous product for the treatment of optic neuritis (January 2025).
Outlook announced results of its NORSE EIGHT study of its Lytenava (bevacizumab gamma) for the treatment of wet AMD (November 2024). The firm also received a positive recommendation from the U.K.’s National Institute for Health and Care Excellence (NICE) for its Lytenava (bevacizumab gamma) for the treatment of wet AMD (December 2024).
Regeneron is continuing to evaluate its higher-dose aflibercept (Eylea HD®) for additional retinal diseases, including macular edema following retinal branch vein occlusion (January 2025).
Roche received approval from the European Medicines Agency for its Vabysmo® (faricimab) 6.0 mg single-dose prefilled syringe for use in the treatment of wet AMD, DME, and macular edema following retinal vein occlusion (RVO, December 2024).
Samsung Bioepis and Biogen received European Commission approval for Opuviz™ (SB15, 40 mg/mL), a biosimilar to aflibercept (November 2024).
SpyGlass Pharma presented data from its first-in-human study of patients with glaucoma or ocular hypertension receiving the SpyGlass IOL-based drug delivery platform (October 2024).
Tenpoint Therapeutics reported results from its BRIO-II study of Brimochol-PF, a fixed-dose combination of brimonidine and carbachol, in the treatment of presbyopia (January 2025). Note that this firm recently merged with Visus, the source of this investigational product (December 2024).
Valo Health presented results of a study of its OPL-0401 rho kinase inhibitor in the treatment of diabetic retinopathy (December 2024).
ViGeneron received clearance from the U.S. FDA for a Phase 1/2 study of its VG801 gene therapy candidate to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene (December 2024).
Viridian Therapeutics announced results from its THRIVE-2 phase 3 clinical trial of veligrotug (veli), an intravenously delivered anti-insulin-like growth factor-1 receptor antibody, in patients with chronic thyroid eye disease (December 2024).
VivaVision announced results from its Phase 2 study of topical VVN461-CS-201, a JAK1/TYK2 inhibitor, for the treatment of post-operative inflammation following cataract surgery (December 2024).
Gene and cell therapy
Adverum Biotechnologies released results from its phase 2 LUNA and OPTIC studies of Ixo-vec (AAV.7m8-aflibercept), a gene therapy for the treatment of wet AMD (November 2024).
BlueRock Therapeutics received US FDA clearance for a Phase 1/2 clinical study of its OpCT-001, a cell therapy for primary photoreceptor diseases (September 2024).
Emmetrope completed its study EMME-001 of EO2002 (magnetic cell nanoparticle delivery system) for the treatment of corneal edema (October 2024).
Lenz Therapeutics and Corxel Pharmaceuticals reported results from a phase 3 trial of its LNZ100 (aceclidine) for the treatment of presbyopia (October 2024).
Nanoscope Therapeutics presented the clinical plan for MCO-010 gene therapy for the treatment of RP (October 2024).
Regenxbio presented results from a phase 2 fellow eye sub-study evaluating the subretinal delivery of ABBV-RGX-314, a gene therapy product, in patients with bilateral wet AMD (October 2024).
Medical devices
Glaukos submitted an NDA to the FDA for Epioxa (Epion), a combination of a cross-linking product and UV-A irradiation, to treat keratoconus with an intact corneal epithelium (December 2024).
Konan listed its objectiveFIELD Analyzer with US FDA (OFA) in late 2024.
LumiThera received authorization from the US FDA for its Valeda Light Delivery System for the treatment of dry AMD (November 2024).
Novoxel received US FDA clearance for its Tixel thermo-mechanical device for the treatment of evaporative dry eye due to meibomian gland dysfunction (MGD, November 2024).
Provectus created a new firm, VisiRose, to develop a Rose Bengal photodynamic antimicrobial therapy for the treatment of infectious keratitis (December 2024).
Regulatory, Government, and Pharmaceutical Industry
AlloVir entered into a merger agreement with Kalaris Therapeutics for the development of retinal therapeutics, including TH103, an anti-vasocative endothelial growth factor fully humanized molecule (November 2024).
Clearside Biomedical’s Asia-Pacific partner, Arctic Vision, entered a commercial collaboration with Santen Pharmaceutical for ARVN001 (suprachoroidal triamcinolone acetonide) (November 2024).
Ocuphire Pharma acquired Opus Genetics (October 2025). The new firm presented results from its Phase 1/2 study of OPGx-LCA5, a gene therapy product to treat LCA5-associated inherited retinal disease, and announced its Phase 3 plan for oral APX3330 for the treatment of severe nonproliferative diabetic retinopathy (December 2024).
One of the major compounding pharmacy firms, Pine Pharmaceuticals, announced its discontinuation of providing aliquoted bevacizumab (off-label) and several other ophthalmic products (October 2024).
Regeneron acquired Oxular, a firm that was developing an injectable dexamethasone, OXU-001 (December 2024).
Ripple Therapeutics entered a business relationship with Glaukos to create sustained-release implants of targeted therapeutics for both glaucoma and retinal diseases (October 2024).
Legal patent proceedings continue by innovator firms against firms developing biosimilar drugs, including a biosimilar of aflibercept (October 2024). Legal proceedings are also ongoing by firms against the federal government regarding the new Medicare 340B rebate model (December 2024).
US Senators Elizabeth Warren (D., Massachusetts) and Josh Hawley (R., Missouri) introduced a bill that would require pharmacy-benefit managers to divest their pharmacy businesses within three years (December 2024).
The US FDA:
In Calendar 2024, approved 50 new molecular entity drugs and 15 new biologicals. This does not include ophthalmic approvals for a novel formulation of clobetasol and three aflibercept biosimilars (December 2024).
Continued enforcement against firms marketing over-the-counter products for illegal inclusion of unlabeled prescription drugs, as well as violations of Good Clinical Practice (November 2024).
Issued a guidance on Gene Therapy Products (November 2024), and, together with the International Conference on Harmonisation, M15 General Principles for Model-Informed Drug Development (January 2025).
Created a program to report new uses for existing drugs, CURE-ID. Among the indications of interest are “long-COVID” (December 2024).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmical, pharmaceutical, and medical device firms. A complete listing of all of G.D.N.’s consulting affiliations can be found online.
Funding Information
No funding was received for this article.