People living with erythropoietic protoporphyria (EPP) often deal with painful skin reactions after even brief light exposure. Afamelanotide is the first drug approved specifically to help extend the amount of pain free light exposure these patients can tolerate.
Key takeaways
- Afamelanotide is approved under the brand name Scenesse for adults with a history of phototoxic reactions from EPP.
- It works as a melanocortin 1 receptor (MC1-R) agonist.
- At least 23 trials have studied afamelanotide, including work in related light sensitivity conditions.
- FAERS reports for afamelanotide are dominated by hypersensitivity type reactions, including anaphylactic reaction and implant site reactions.
- The label does not carry a boxed warning.
What Afamelanotide Is
Afamelanotide is a synthetic peptide that acts on the melanocortin 1 receptor (MC1-R), a receptor involved in skin pigmentation pathways. Its identifier in PubChem is CID 16197727. By engaging this receptor, the compound is designed to help the skin build tolerance to light exposure.
What Afamelanotide Is Approved For
Afamelanotide, marketed as Scenesse, is indicated to increase pain free light exposure in adult patients who have a history of phototoxic reactions caused by erythropoietic protoporphyria (EPP). This is a light sensitivity disorder in which skin reacts painfully to sunlight. The approved label does not include a boxed warning.
What the Evidence Shows
Afamelanotide has been evaluated across at least 23 registered clinical trials. A completed Phase 3 trial examined afamelanotide in patients with polymorphic light eruption (PLE), another light sensitivity condition. Separate Phase 2 trials have looked at afamelanotide in variegate porphyria and in xeroderma pigmentosum, with the variegate porphyria study completed and the xeroderma pigmentosum study status listed as unknown.
Reported Side Effects
Reports submitted to FAERS for afamelanotide are report counts, not confirmed incidence or proof of causation. The most frequently reported reactions involve hypersensitivity, including anaphylactic reaction (4 reports), type I hypersensitivity (3), and contraindication to medical treatment (3). Several reports describe reactions at the implant site itself, such as implant site hypersensitivity, pruritus, and urticaria (2 reports each). Vomiting (2 reports) and acute kidney injury (1 report) were also noted.