Q1 2025 Recap: Ophthalmology News and Updates

Ophthalmology in Q1 2025 saw pivotal advancements across the regulatory, clinical, and innovation landscapes. Updates from the US Food and Drug Administration (FDA) marked progress in treatment delivery, long-term disease management, and first-in-class approvals, while late-stage trial data signaled shifting paradigms for chronic retinal diseases and neuroprotective strategies.

The ophthalmic field continues to gain momentum, with sustained industry interest in extending durability, reducing treatment burden, and addressing previously untreatable conditions. Regulatory wins were matched by major trial readouts, but also included notable setbacks, reflecting the evolving rigor of FDA expectations.

This quarter, the HCPLive Ophthalmology team covered 5 major FDA regulatory updates, 5 headline trial developments, and 3 expert-driven insights, including two episodes of New Insight highlighting landmark therapies for presbyopia and macular telangiectasia type 2 (MacTel).

Q1 2025 Regulatory Updates in Ophthalmology

FDA Approves Susvimo for Diabetic Macular Edema

On February 4, 2025, the FDA approved Susvimo (ranibizumab injection) 100 mg/mL for intravitreal use via ocular implant as the first continuous delivery treatment for diabetic macular edema (DME). The approval marked the second indication for Genentech’s Susvimo after its initial approval in 2021 for neovascular (wet) age-related macular degeneration (nAMD).

FDA Expands Dosing Label for Avacincaptad Pegol for Geographic Atrophy

On February 13, 2025, the FDA approved the expanded label for avacincaptad pegol intravitreal solution (IZERVAY) for the treatment of geographic atrophy (GA), extending the approved dosing beyond 12 months. The decision was made after the supplemental NDA was resubmitted by Astellas Pharma in December 2024, after initial feedback provided by the FDA the month prior.

FDA Accepts NDA for Epioxa for Keratoconus Treatment

On February 24, 2025, the FDA accepted the New Drug Application for Epioxa (Epi-on), a corneal cross-linking therapy for keratoconus, a sight-threatening corneal disease. The regulatory agency set a Drug User Fee Act (PDUFA) target action date of October 20, 2025, reflecting a standard 10-month review period.

FDA Approves Revakinagene Taroretcel (ENCELTO) for MacTel

On March 6, 2025, the FDA approved revakinagene taroretcel-lwey (ENCELTO), formerly known as NT-501, for Macular Telangiectasia type 2 (MacTel). Neurotech Pharmaceuticals’ approval marked the first and only FDA–approved treatment for MacTel, a neurodegenerative disease of the retina that can cause progressive and irreversible vision loss.

FDA Issues Complete Response Letter to Reproxalap for Dry Eye Disease

On April 3, 2025, the FDA issued a Complete Response Letter (CRL) to Aldeyra Therapeutics for the resubmission of the NDA reproxalap, a topical ocular treatment for dry eye disease (DED). The regulatory agency cited a failure to demonstrate efficacy in adequate and well-controlled studies, expressing concern that a recent trial may have influenced interpretation of the final results.

Q1 2025 Trial Announcements in Ophthalmology

OCS-05 for Acute Optic Neuritis Achieves Key Endpoints in Phase 2 Trial

OCS-05 met its key primary safety endpoint and achieved statistical significance on efficacy-based secondary endpoints for acute optic neuritis in the Phase 2 ACUITY trial. Oculis’ investigational neuroprotective therapy achieved a favorable safety and tolerability profile, with neuroprotective structural benefit and capability to improve visual function.

Aflibercept 8 mg for RVO Hits Primary Endpoint in Phase 3 QUASAR Trial

Aflibercept 8 mg (EYLEA HD) met its primary endpoint in the Phase 3 QUASAR trial with improved, noninferior vision gains with extended dosing versus aflibercept 2 mg in patients with macular edema following retinal vein occlusion (RVO). Regeneron announced plans to submit a supplementary Biologics License Application (sBLA) to the FDA in early 2025.

4D-150 Reduces Injection Burden in DME, Paving Way for FDA-Backed Phase 3

Topline interim data from the SPECTRA clinical trial showed the positive safety and clinical activity for 4D-150 in patients with diabetic macular edema (DME), with sustained gains in best-corrected visual acuity (BCVA) and a reduction in central subfield thickness (CST). 4DMT announced alignment with the FDA on a single Phase 3 clinical trial as the basis for the 4D-150 Biologics License Application (BLA) submission in DME.

DURAVYU Hits Primary Phase 2 Endpoint, Reduces Treatment Burden in DME

Six-month data from the Phase 2 VERONA trial assessing the vorolanib intravitreal insert (DURAVYU) showed a meaningful improvement in vision and anatomy in DME, meeting the trial’s primary and secondary endpoints. EyePoint Pharmaceuticals reported the 1.34 mg and 2.7 mg doses achieved an extended time to the first supplemental anti-VEGF injection, compared with the aflibercept control cohort.

Opthea Announces Termination of ShORe and COAST Trials of Sozinibercept

Sozinibercept, a novel vascular endothelial growth factor (VEGF) “trap” fusion protein intended to treat wet AMD, failed to demonstrate the vision improvements required for success in the Phase 3 COAST trial. Opthea has decided to terminate both the COAST and ShORe trials and cease the development of sozinibercept in wet AMD.

Q1 2025 Expert Perspectives in Ophthalmology

Examining the Role of Advocacy in Ophthalmology with Michael X. Repka, MD

In a recent interview with HCPLive, Michael X. Repka, MD, a professor of ophthalmology and pediatrics at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, and 2025 President of the American Academy of Ophthalmology, outlined the critical role of advocacy in shaping ophthalmologic policies and ensuring equitable patient care. Repka emphasized the need for ongoing legislative and regulatory engagement to secure adequate vision and eye health resources amidst competing healthcare priorities.

New Insight: A Look at Qlosi for Presbyopia with Elad Kedar, MBA

In this episode of New Insight, host Veeral Sheth, MD, MBA, director of clinical research at University Retina and Macula Associates, explored the approval and impending launch of Qlosi (pilocarpine HCL ophthalmic solution) 0.4% for managing presbyopia, with Elad Kedar, MBA, chief executive officer of Orasis Pharmaceuticals.

New Insight: Exploring ENCELTO for MacTel with Rich Small, CEO of Neurotech

Another episode of New Insight explored the historic FDA approval of revakinagene taroretcel-lwey (ENCELTO) for MacTel with Rich Small, chief executive officer of Neurotech.