IPM Take
This is exactly where eye-disease innovation hits the access wall. In geographic atrophy, a treatment may slow lesion growth, but patients, regulators and payers still need to know whether that translates into vision people can use in daily life. EMA’s workshop matters because it moves the debate from “does the scan look better?” to “does the patient see, function or live better?” That distinction will shape whether Europe can approve, reimburse and deliver future GA therapies.
Executive Summary
On 29 April 2026, the European Medicines Regulatory Network held a workshop on geographic atrophy endpoints. EMA describes geographic atrophy as a slowly progressing, seriously debilitating condition with high prevalence, unmet medical need and major effects on patients and families. The workshop focused on the need to show clinically relevant treatment benefit on visual function to support centralised marketing authorisation, and on whether structural imaging endpoints can robustly predict patient-relevant benefit. The discussion follows a difficult European regulatory context, including EMA’s confirmed refusal of marketing authorisation for Syfovre, pegcetacoplan, after concluding that reduced lesion growth did not translate into clinically meaningful benefit for patients.
Why it matters
- Regulators: Need endpoint standards that can evaluate whether imaging or structural retinal changes predict meaningful visual benefit.
- HTA bodies / payers: Will need evidence that future GA therapies improve outcomes patients value, not only biomarker or imaging measures.
- Patients / advocates: Can push for trial endpoints that reflect daily functioning, independence and lived experience with progressive vision loss.
Before this workshop, the GA debate was already tense. Trials often measure structural change, such as lesion growth, while patients and regulators want evidence that treatment preserves meaningful visual function. In Europe, this issue became highly visible after EMA refused Syfovre, despite GA therapies being available in other markets.
What has changed is that EMA is directly convening the endpoint question. The workshop aims to clarify regulatory expectations for endpoints, identify research gaps and discuss how structural changes and retinal function relate to patient benefit. EMA’s agenda states that alternative primary endpoints, including retinal imaging-based endpoints, must be able to robustly predict clinically relevant benefit for patients within a reasonable period after treatment.
The affected population is adults with geographic atrophy secondary to age-related macular degeneration, a condition that can progressively damage central vision and daily functioning. The implementation implication is sharp. If Europe cannot agree on credible endpoints, future GA therapies may face the same access uncertainty: promising biological effect, but unclear patient-relevant value.
For IPM, GA is a warning for precision eye care more broadly. Imaging biomarkers are powerful, but reimbursement and adoption will depend on evidence that patients, clinicians and payers recognise as meaningful.
