Erythropoietic protoporphyria photoprotection
Langendonk 2015 (NEJM, n=168) randomized EPP patients to subcutaneous afamelanotide implants vs placebo across two multicenter trials and showed significantly increased duration of sun exposure without pain and improved quality of life. This led to EMA approval (2014) and FDA approval (2019) — the only melanocortin agonist with a fully approved adult indication.
4 human studies · 6 animal studies
Key findings & citations
- Langendonk 2015: significant increase in pain-free sun exposure time vs placebo over 270 days.
- Quality of life improvements consistent across European and US populations.
- EMA and FDA approved (Scenesse, Clinuvel) for adult EPP.
Top citations
Our take
Real, niche indication. Approved drug with clean evidence — but EPP affects fewer than 1 in 75,000, so this isn't relevant to most users.