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Afamelanotide: A Review in Erythropoietic Protoporphyria


Afamelanotide (SCENESSE®) is a synthetic α-melanocyte stimulating hormone analogue and first-in-class melanocortin-1 receptor agonist that is approved in the EU for the prevention of phototoxicity in adults with erythropoietic protoporphyria (EPP). It is administered subcutaneously as a biodegradable, controlled-release implant containing 16 mg of afamelanotide. This article reviews the clinical efficacy and tolerability of afamelanotide in EPP and summarizes its pharmacological properties. In the phase III trial, CUV039, afamelanotide treatment improved light tolerance in patients with EPP. Compared with placebo, afamelanotide treatment enabled patients to spend more time in direct sunlight without pain and increased the time to the appearance of the first symptoms of phototoxicity provoked by a standardized light source. Afamelanotide was generally well tolerated in this trial, with no drug-related serious adverse events reported. Commonly occurring adverse reactions included headache and implant-site reactions. Efficacy and safety data from earlier phase III trials are consistent with those from the CUV039 trial. Afamelanotide, approved in the EU for the prevention of EPP phototoxicity, represents a useful addition to the management of the disorder.

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During the peer review process, the manufacturer of afamelanotide was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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Corresponding author

Correspondence to Esther S. Kim.

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The preparation of this review was not supported by any external funding.

Conflicts of interest

Esther S. Kim and Karly P. Garnock-Jones are salaried employees of Adis/Springer, are responsible for the article content and declare no relevant conflicts of interest.

Additional information

The manuscript was reviewed by: K. E. Anderson, The Departments of Preventive Medicine and Community Health, University of Texas Medical Branch, Galveston, TX, USA; H. L. Bonkovsky, Department of Internal Medicine, School of Medicine, Wake Forest University, Winston-Salem, NC, USA; J. G. Langendonk, Department of Internal Medicine, Center for Lysosomal and Metabolic Diseases, Netherlands Porphyria Center, Erasmus MC, Rotterdam, Netherlands; H. W. Lim, Department of Dermatology, Henry Ford Hospital, Detroit, MI, USA; L. E. Rhodes, The Centre for Dermatology, University of Manchester, Salford Royal Hospital, Manchester, UK.

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Kim, E.S., Garnock-Jones, K.P. Afamelanotide: A Review in Erythropoietic Protoporphyria. Am J Clin Dermatol 17, 179–185 (2016).

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