For people with erythropoietic protoporphyria (EPP), even brief sun exposure can trigger severe pain. Afamelanotide is the first approved option designed to extend the amount of light a person with EPP can tolerate without that reaction.
Key takeaways
- Afamelanotide is approved under the brand name Scenesse for adult patients with erythropoietic protoporphyria (EPP).
- It works as a melanocortin 1 receptor (MC1-R) agonist.
- It is indicated to increase pain free light exposure in people with a history of phototoxic reactions from EPP.
- At least 23 trials have studied afamelanotide, including work in vitiligo and as an adjunct to photodynamic therapy.
- FAERS reports linked to afamelanotide are dominated by hypersensitivity-type reactions, including anaphylaxis and implant site reactions.
What afamelanotide is
Afamelanotide is a peptide compound (PubChem CID 16197727) that acts on the melanocortin 1 receptor, known as MC1-R. By engaging this receptor, it is designed to help the skin build tolerance to light exposure in people prone to phototoxic reactions.
What afamelanotide is approved for
Afamelanotide is approved, under the brand name Scenesse, to increase pain free light exposure in adult patients who have a history of phototoxic reactions from erythropoietic protoporphyria. EPP is a condition in which exposure to light causes significant pain, and this approval targets that specific burden. No boxed warning is listed on the label.
What the evidence shows
At least 23 registered trials have evaluated afamelanotide across different populations. A completed Phase 3 safety extension study followed patients with EPP over time. Separately, completed Phase 2 studies examined afamelanotide combined with narrow-band UVB light for nonsegmental vitiligo and as adjunctive therapy alongside photodynamic therapy using porfimer sodium.
Reported side effects
Reports submitted to FAERS for afamelanotide are report counts, not confirmed rates or causation, but they point to a hypersensitivity-related pattern. The most frequently reported terms involve allergic-type reactions, including anaphylactic reaction (4 reports) and type I hypersensitivity (3). Several reports describe reactions localized to the implant site, such as hypersensitivity, pruritus, and urticaria (2 reports each). Vomiting and malignant melanoma were each reported twice.