Study summary · research use only
Afamelanotide: An Orphan Drug with Potential for Broad Dermatologic Applications
Plain-language summary
Paraphrased from the published abstract below — not a verdict on whether anything works.
This review discusses afamelanotide (SCENESSE), a synthetic α-melanocyte-stimulating hormone analogue approved to increase pain-free sunlight exposure in adult patients with erythropoietic protoporphyria, and its potential dermatologic applications. The authors searched PubMed/MEDLINE and ClinicalTrials.gov for literature and trials on afamelanotide in cutaneous disease, reporting randomized controlled trial evidence for its use beyond erythropoietic protoporphyria in polymorphic light eruption and vitiligo, and smaller studies describing use in acne vulgaris, Hailey-Hailey disease, and solar urticaria. The authors report no serious adverse events with afamelanotide, though diffuse hyperpigmentation is experienced by almost all patients, and state larger studies are needed to confirm its role in dermatologic conditions beyond erythropoietic protoporphyria, with further research needed on dosing in pediatric patients.
Abstract
Afamelanotide (SCENESSE®) is a synthetic analogue of α-melanocyte-stimulating hormone that is FDA-approved to increase pain-free sunlight exposure in adult patients with erythropoietic protoporphyria. Its dual photoprotective and anti-inflammatory effects also make it a promising therapy for other photosensitive dermatologic diseases that are resistant to treatment. The PubMed/MEDLINE and ClinicalTrials.gov databases were searched for literature and ongoing trials describing the use of afamelanotide in treating cutaneous diseases. There is randomized controlled trial (RCT) evidence for the successful use of afamelanotide in several conditions beyond erythropoietic protoporphyria, including polymorphic light eruption and vitiligo. Smaller studies have also demonstrated its efficacy in treating acne vulgaris, Hailey-Hailey disease, and solar urticaria. No serious adverse effects with afamelanotide use have been reported, though diffuse hyperpigmentation is experienced by almost all patients. Larger scale studies are needed to confirm the efficacy of afamelanotide in treating dermatologic conditions beyond erythropoietic protoporphyria, and further research should focus on determining the safety, efficacy, and optimal dosing of afamelanotide for pediatric patients.J Drugs Dermatol. 2021;20(3):290-294. doi:10.36849/JDD.5526.
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