Abstract
In this review period, results from a pivotal study of an oral treatment for Stargardt’s Disease were reported. Enrollment was completed in several pivotal studies for novel therapies in a range of ocular diseases. Controversies continue with the U.S. Food and Drug Administration (FDA) with respect to execution of novel review programs and the requirements for approval of novel therapies, especially for rare or life-threatening diseases.
Ophthalmic Pharmaceuticals and Biologics
Belite Bio reported results of a Phase 3 trial of its oral tinlarebant for the treatment of Stargardt disease type 1 and has begun submission of a New Drug Application (NDA, April/May 2026). Cambium Bio announced its plans to conduct a single pivotal clinical study, together with confirmatory evidence, for its development of Elate Ocular® (fibrinogen-depleted, allogeneic human platelet lysate) for the treatment of moderate-to-severe dry eye disease (DED, April 2026). Eclipse Life Sciences completed patient enrollment in its BETTIS-1 phase 2 clinical trial evaluating its EC-104 fluocinolone acetonide extended release for the treatment of diabetic macular edema (DME, April 2026). Genentech gained approval for the secondary indication of treatment of macular edema due to retinal vein occlusion for its Vabysmo® (faricimab, April 2026). Kubota Vision is continuing to develop its emixustat hydrochloride for the treatment of macular atrophy secondary to Stargardt disease (May 2026). Lenz Therapeutics submitted a marketing application in the United Kingdom for its Vizz® (aceclidine) for the treatment of presbyopia (April 2026). Oculis completed the final patient visit in its DIAMOND phase 3 program assessing OCS-01 (topical dexamethasone) in patients with DME (April 2026). The firm also received a Special Protocol Assessment agreement from the U.S. FDA for its Phase 3 PIONEER-1 trial of systemic privosegtor in optic neuritis (May 2026). Viridian announced data from the REVEAL-2 phase 3 clinical trial of subcutaneous elegrobart in patients with chronic thyroid eye disease (May 2026).
Drug Delivery
Ocular Therapeutix reported additional data from its SOL-1 Phase 3 Trial of its Axpaxli™ in the treatment of wet age-related macular degeneration. The firm also announced completion of enrollment in an extension study (April 2026).
Gene and Cell Therapy
Lineage Cell Therapeutics announced 36-month data from its phase 1/2a clinical study of its RG6501 (OpRegen), retinal pigment epithelium cell therapy, for the treatment of geographic atrophy (GA, May 2026). MeiraGTx re-acquired from Johnson & Johnson* (J&J) rights to the gene therapy botaretigene sparoparvovec bota-vec for the treatment of X-linked retinitis pigmentosa (April 2026).
Regulatory, Government, and Pharmaceutical Industry
Bayer will acquire Perfuse Therapeutics, including PER-001, a small molecule intravitreal endothelin receptor antagonist currently in Phase 2 clinical development for the treatment of glaucoma and diabetic retinopathy (May 2026). The European Medicines Agency selected three optic neuritis treatments for its “PRIME” PRIority MEdicines designation (privosegtor, rapcabtagene autoleucel, and puliretgene parvec, April 2026). There is a growing proportion of prescription medications that are being paid for by patients out-of-pocket (OOP), and thus not accounted for in state-run prescription drug monitoring programs. Mattingly proposes expanding these important public health programs to include OOP medications.
1
The U.S. FDA:
Proposed that an approved drug be withdrawn based upon a claim that the Sponsor’s data contained untrue statements of material fact (April 2026). Announced the real-time clinical trials program, which allows for rolling clinical development (i.e., Phase 1/2/3, April 2026). Issued a Complete Response Letter (i.e., nonapproval) to Replimune for its Biologics License Application for RP1 (vusolimogene oderparepvec) in combination with nivolumab for the treatment of advanced melanoma (April 2026). As requested by Sanofi, removed its application for an additional indication for its diabetes drug Tzield™ (teplizumab) from the FDA Commissioner’s National Priority Voucher program. This appears related to controversies within the FDA’s Center for Drug Evaluation and Research (April 2026).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic, pharmaceutical, and medical device firms.
Funding Information
No funding was received for this article.