Durability and earlier access could reshape diabetic retinopathy treatment

This year’s World Sight Day turns global attention to the scale of preventable vision loss. Diabetic retinopathy (DR) is the leading cause of blindness among working-age adults. An estimated 9.6 million Americans were living with the disease in 2021, including 1.84 million with vision-threatening disease, figures that have roughly doubled since 2004 as diabetes climbed past 37 million and average glycaemic control worsened.

Yet, the treatment paradigm remains strikingly cautious. In our recent study of 103 US retinal specialists and general ophthalmologists, for example, respondents reported that only about half of their patients receive intravitreal therapy, that biologic use concentrates at the more advanced end of the disease, and that initiation is overwhelmingly reactive, driven by an effort to prevent progression or by emerging vision complications.

The most common reason candidates go untreated is simply that their disease is not considered severe enough. The majority of these patients fall into the mild-to-moderate non-proliferative categories – a large and underserved pool that represents both the disease's greatest commercial opportunity and its most contested clinical frontier.

A reactive paradigm with a clinical rationale

This restraint is not inertia; it reflects the evidence. The NEI-funded DRCR Retina Network Protocol W trial found that preventive aflibercept in moderate-to-severe non-proliferative disease reduced progression to proliferative retinopathy and centre-involved macular oedema, but produced no visual-acuity benefit at two or four years. The investigators concluded that monitoring, with treatment only as needed, remained the sound approach.

The result is a large pool of clinically eligible but untreated patients, alongside real appetite for agents that would justify earlier and more durable intervention. Asked about the greatest unmet needs, respondents converged on durability, lower injection burden, and non-invasive options, with more than one describing current therapies as band-aids, rather than a cure.

Access defines the current market more than clinical preference

The distance between preference and practice is about access. Step-through requirements apply most of the time, insurance and reimbursement rank as the single largest barrier to biologic selection. Roughly half of respondents call the prior authorisation process a high burden, with the resulting outcome of patients facing blocks in moving towards more efficacious options.

These access hurdles result in a gap between what physicians use and what they prefer. Bevacizumab (Avastin), used off-label, still dominates share and new prescriptions, propelled by habit and cost, yet, it carries the weakest perception in the class, including a net promoter score of negative 26. Given a free choice without payer constraints, 60% of respondents instead named high-dose aflibercept, marketed as Eylea HD, which earned the highest satisfaction and net promoter score on the strength of its durability.

Innovation is breaking the injection template

For two decades, treating diabetic retinopathy has meant repeated anti-VEGF injections. The late-stage pipeline breaks from that template on the axes that matter most, offering novel mechanisms, novel delivery, and durability measured in months or years, rather than weeks. Several candidates act upstream of existing agents, targeting the inflammation and signalling behind early microvascular damage, rather than neutralising VEGF after it forms.

Generating the most enthusiasm is a one-time gene therapy. Sura-vec (ABBV-RGX-314), from AbbVie and RegenxBio, uses an in-office suprachoroidal injection to deliver sustained anti-VEGF expression, and respondents rated it the greatest perceived advance and the agent they would most like to see approved. The programme advanced in 2026 into the pivotal Phase 2b/3 NAAVIGATE trial, with 3-year ALTITUDE follow-up presented in [September 2026].

Aiming to reduce the treatment burden with longer dosing intervals, is Axpaxli (OTX-TKI) from Ocular Therapeutix, a sustained-release axitinib hydrogel designed for once-yearly dosing in diabetic retinopathy. It drew the highest aided awareness among surveyed physicians, and its registrational HELIOS-3 trial in non-proliferative disease began enrolling in late 2025. Kodiak Sciences' anti-VEGF conjugate tarcocimab tedromer (proposed brand name Zenkuda) takes a similar route, with dosing as infrequent as every six months.

The rest of the pipeline offers something other than durability: a different route or a different mechanism. One of the most anticipated agents is the oral Oruvestat (VX-01) from Vantage Biosciences, which targets the neurovascular inflammation of early disease. Respondents assigned it the highest anticipated peak share of any pipeline agent, a measure of how strongly the oral route resonates. As one physician noted, "Nobody likes injections and everyone understands pills." Its Phase 2 SeeClear study completed enrolment in August 2026, with data due in late 2027. Boehringer Ingelheim's anti-Sema3A antibody BI 764524 brings a novel mechanism of its own.

Innovation is not only in the vial

Innovation extends beyond therapeutics. Physicians describe the disease as silent early on, and our data shows, for example, that most patients reach a retina specialist only by referral, most often from optometry and increasingly from primary care and endocrinology. That is where autonomous artificial intelligence is reshaping the field.

Several systems, including LumineticsCore (formerly IDx-DR), EyeArt, and AEYE-DS, now hold FDA clearance to detect more-than-mild disease from fundus images without a clinician reading the scan, and are increasingly installed in primary care, endocrinology, and community health settings. By moving detection upstream to where people with diabetes already receive care, these tools address one of the clearest unmet needs respondents named.

Durability will matter most when access supports earlier use

The opportunity emerging from these strands of research is to align earlier intervention with treatments that offer sustained disease control. However, that benefit may be limited if step edits keep a therapy in later-line use, particularly when a patient's disease is not adequately controlled on the required initial treatment. Retinal damage can continue to accumulate while access to an appropriate alternative is delayed.

Manufacturers will need to demonstrate an improvement over existing care and translate that evidence into a commercial access strategy that supports earlier use. Addressing step requirements should therefore be integral to planning for therapies intended to change when and how DR is treated.

Manufacturers that pair meaningful clinical improvement with access at the point of need will be best positioned to reshape DR treatment. Combined with screening and timely referral, this could help ophthalmologists intervene before vision is lost. If the data supports their promise, the therapies now in trials could enable earlier, more durable disease control and reduce reliance on treatment that begins only after complications emerge.

About the author

Sarah Soucy is franchise head, ophthalmology, at Spherix Global Insights. Soucy is a healthcare market research professional with over 10 years of experience in pharmaceutical insights and analytics. Throughout her career, she has held leadership roles at RxY, REACH Market Research, and Clarivate. She began her professional journey as a scientist within Pfizer's inflammation and immunology groups before transitioning into market research. Today, she applies that foundational scientific rigour to deliver deep, actionable insights for clients in ophthalmology.