Abstract
In this review period, two ophthalmic drugs were approved: one a new chemical entity and new mechanism and the other a fixed-dose combination of autonomic drugs long used in ophthalmology. New molecules and novel gene therapies continue to be evaluated in retinal diseases. Ophthalmic firms are using US FDA pathways for accelerated development and review. The US FDA continues to issue guidance documents and policy statements suggesting decreased regulation.
Ophthalmic Pharmaceuticals and Biologics
Kalaris announced data from its phase 1a single ascending dose trial of TH103, a fully humanized, recombinant fusion protein that acts against Vascular Endothelial Growth Factor (VEGF) as a decoy receptor, in treatment-naïve patients with neovascular age-related macular degeneration (AMD, December 2025). Novaliq received clearance from the US FDA for an Investigational New Drug (IND) application for its NOV05 (tacrolimus) for topical treatment of noninfectious anterior uveitis (January 2026). Oculis received Breakthrough Therapy Designation by the US FDA for its privosegtor for the treatment of optic neuritis (January 2026). OKYO received US FDA authorization for compassionate use of its urcosimod for neuropathic pain (January 2026). Ollin Biosciences announced results from its randomized, head-to-head phase 1b JADE clinical study evaluating OLN324, a VEGF/Ang2 bispecific antibody, versus faricimab (Vabysmo®) in subjects with diabetic macular edema or wet AMD (January 2026). Senju received approval in Japan for its Avarept® Ophthalmic Suspension (Motugivatrep, SJP-0132) for the treatment of dry eye disease. Motugivatrep is a transient receptor potential cation channel V subfamily V antagonist (December 2025). Spyglass Pharma randomized subjects into phase 3 trials of its bimatoprost-drug pad intraocular lens system product (January 2026). TenPoint received US FDA approval for its Yuvezzi® (carbachol and brimonidine tartrate ophthalmic solution for the treatment of presbyopia) (January 2026). TheiaNova dosed a subject in a trial of its topical ocular combination drug (TGF-β3 and dexamethasone) for the treatment of keratoconus (January 2026).
Gene and Cell Therapy
Complement Therapeutics received Fast Track designation from the US FDA for CTx001, its gene therapy candidate for the treatment of geographic atrophy (GA) secondary to AMD (January 2026). Ocugen announced preliminary results from its phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), a gene therapy for GA secondary to dry AMD (January 2026). PulseSight Therapeutics completed enrollment in a phase 1 clinical trial of its PST-611, a gene therapy for transferrin, for the treatment of dry AMD and GA (January 2026).
Medical Devices
Allotex’s investigational device exemption for its human collagen-based corneal implant was cleared by the US FDA for use in subjects with presbyopia (January 2026).
Regulatory, Government, and Pharmaceutical Industry
Amgen received a request from the US FDA to remove its treatment for antineutrophil cytoplasmic autoantibody-associated vasculitis, Tavneos® (avacopan), from the market. The request was based upon assessment of 9/331 patients in a phase 3 trial (used in the 2021 approval) and a concern about benefit/risk. The firm did not remove the product, stating that they were not aware of any issues with patient data (January 2026). China’s 2015 reform of their regulation of drugs by the China National Medical Products Administration (NMPA) has reduced the lag for geriatric drug approval relative to Europe and the United States, but there is still a lag.
1
Since that reform, the NMPA approved 167 innovative drugs, 58% of which were small molecules, and 87% of which were domestic applications.
2
Everyone Medicines received clearance from the UK Medicines and Healthcare products Regulatory Agency to treat a patient with Niemann–Pick disease with an antisense oligonucleotide under a new regulatory program (January 2026). Kubota Holdings is working with Laboratoires KÔL for a license for its emixustat for the treatment of retinal diseases (December 2025). Small pharmaceutical firms experience the same US FDA regulatory success as larger firms in the period of 2015–2022 (January 2026).
3
The US divestment in federal support of scientists is causing a loss in the number of scientists, in contrast to the investment by China, according to articles in Science magazine.4,5 US FDA:
Issued guidance on: clinical decision support software, and Bayesian biostatistics (January 2026). Made policy statements on: increased flexibility on requirements for cell and gene therapies and increased the scope of products that are considered “General Wellness,” which do not require premarket review (January 2026). The European Medicines Agency provided 104 positive opinions in 2025 on drugs submitted. Of these, 38 were new active substances. Of these, the ophthalmic drugs included Tepezza® (teprotumumab-trbw) for the treatment of thyroid eye disease, Ryjunea® (atropine) for slowing pediatric myopic progression,
6
and several biosimilars of aflibercept and ranibizumab (January 2026). The US National Institutes of Health announced a policy limiting funds available to pay authors’ open-access publishing fees,
7
potentially placing authors in a challenging situation given the increasing costs from publishers.
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic, pharmaceutical, and medical device firms.
Funding Information
No funding was received for this article.