title: Afamelanotide: A Review in Erythropoietic Protoporphyria.
abstract: 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.
authors.affiliation: Springer, Private Bag 65901, Mairangi Bay 0754, Auckland, New Zealand. demail@springer.com.
authors.forename: Esther S
authors.initials: ES
authors.lastname: Kim
authors.affiliation: Springer, Private Bag 65901, Mairangi Bay 0754, Auckland, New Zealand.
authors.forename: Karly P
authors.initials: KP
authors.lastname: Garnock-Jones
mesh_terms: D020341:Absorbable Implants / Q000009:adverse effects; D000328:Adult; D017326:Clinical Trials, Phase III as Topic; D017484:Dermatitis, Phototoxic / Q000517:prevention & control*; D004343:Drug Implants / Q000008:administration & dosage / Q000009:adverse effects; D005062:European Union; D006261:Headache / Q000209:etiology; D006801:Humans; D046351:Protoporphyria, Erythropoietic / Q000188:drug therapy*; D044102:Receptor, Melanocortin, Type 1 / Q000819:agonists*; D065566:Subcutaneous Absorption; D013472:Sunlight / Q000009:adverse effects; D016896:Treatment Outcome; D000521:alpha-MSH / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use
chemical_list: D004343:Drug Implants; D044102:Receptor, Melanocortin, Type 1; D000521:alpha-MSH; C534526:afamelanotide