Peptide Graded

Afamelanotide

Does Afamelanotide work?

Yes, in large controlled trials: two trials in 168 adults.

AStrong human evidenceFDA approvedas of 2026-10-26

Two randomized, double-blind, placebo-controlled trials in 168 adults with EPP (94 in the United States, 74 in the European Union) showed more pain-free hours in sunlight, and a 117-patient post-authorisation cohort followed for about two years confirmed the effect in routine practice. Consensus guidelines recommend it for preventing phototoxic symptoms. The evidence is strong for EPP and nonexistent for the tanning use the compound is also known for. 567

Two multicentre randomized trials were published together in 2015. They enrolled 168 adults with EPP. The US study had 94 patients and ran 270 days. Median pain-free time in direct sunlight was 69.4 hours with afamelanotide. With placebo it was 40.8 hours. The European Union study had 74 patients and ran 180 days. There the figures were 6.0 hours versus 0.8 hours. Phototoxic reactions were also fewer (77 versus 146). Quality of life got better in both trials. 5

A cohort study was run at Erasmus MC after approval. It followed 117 EPP patients on afamelanotide for a median of two years. Time outdoors rose by 6.1 hours per week. Quality of life scores for the disease got about 14% better. Reactions were less painful, though not fewer. 98% of patients stayed on treatment. 6

Evidence checklist

  • Randomised controlled trial Two placebo-controlled trials in EPP
  • Human study over 100 people 168 trial patients; 117-patient cohort
  • More than one research group US and EU trials plus Dutch academic cohort
  • Clinical outcomes, not surrogate markers Pain-free sun time, phototoxic reactions
  • Human safety data beyond case reports Trials, two-year cohort and label data

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More on Afamelanotide

Citations

  1. FDA: Scenesse (afamelanotide) prescribing information, 2019 accessdata.fda.gov
  2. McGill Office for Science and Society: the 'Barbie drug' mcgill.ca
  3. EMA: Scenesse European public assessment report ema.europa.eu
  4. BioSpace 2019: FDA approves first treatment to increase pain-free light exposure in EPP biospace.com
  5. Langendonk 2015, NEJM: afamelanotide for erythropoietic protoporphyria pubmed.ncbi.nlm.nih.gov
  6. Wensink 2020, JAMA Dermatology: afamelanotide outcomes in clinical practice pubmed.ncbi.nlm.nih.gov
  7. J Am Acad Dermatol 2023: consensus guidelines for EPP and X-linked protoporphyria pubmed.ncbi.nlm.nih.gov
  8. DailyMed: Scenesse label, warnings and precautions dailymed.nlm.nih.gov
  9. Langendonk 2015, NEJM (DOI) doi.org

Reference information only, not medical advice. Drawn from the Afamelanotide article, fact-checked against the cited sources. No dosing anywhere on this site.