Springer, Private Bag 65901, Mairangi Bay 0754, Auckland, New Zealand.
Am J Clin Dermatol. 2016 Apr;17(2):179-85. doi: 10.1007/s40257-016-0184-6.
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.
阿法美拉汀（SCENESSE(®)）是一种合成的α-促黑素细胞激素类似物，也是首个获批上市的黑素皮质素 1 受体激动剂，在欧盟被批准用于预防红细胞生成性原卟啉症（EPP）成人患者的光毒性。它以含有 16mg 阿法美拉汀的可生物降解、控释植入剂的形式皮下给药。本文综述了阿法美拉汀治疗 EPP 的临床疗效和耐受性，并总结了其药理学特性。在 CUV039 期 3 临床试验中，阿法美拉汀治疗改善了 EPP 患者的光耐受性。与安慰剂相比，阿法美拉汀治疗使患者能够在没有疼痛的情况下在阳光下停留更长时间，并增加了出现由标准光源引发的光毒性的第一个症状的时间。在这项试验中，阿法美拉汀总体上具有良好的耐受性，没有报告与药物相关的严重不良事件。常见的不良反应包括头痛和植入部位反应。早期的期 3 临床试验的疗效和安全性数据与 CUV039 试验一致。阿法美拉汀在欧盟被批准用于预防 EPP 光毒性，它是治疗该疾病的一种有用方法。
