Afamelanotide for treating erythropoietic protoporphyria

3 The technology

3.1 Afamelanotide (Scenesse, Clinuvel) activates the synthesis of eumelanin mediated by the MC1R receptor. Eumelanin contributes to photoprotection by: strongly absorbing UV and visible light (acting as a filter); antioxidant activity; and inactivating the superoxide anion and increasing the availability of superoxide dismutase to reduce oxidative stress. Afamelanotide has a marketing authorisation in the UK under 'exceptional circumstances' for the 'prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP)'. It is administered as a subcutaneous dissolving implant. One implant is administered every 2 months before expected and during increased sunlight exposure, for example, from spring to early autumn. The marketing authorisation recommends 3 implants per year, depending on the length of protection needed. The maximum number of implants recommended in the marketing authorisation is 4 per year. The marketing authorisation stipulates that afamelanotide should only be prescribed by specialist clinicians in recognised porphyria centres, and that it should only be given by a clinician trained and accredited by the marketing authorisation holder to insert the implants.

3.2 The most common side effects with afamelanotide seen in clinical trials were nausea and headache, and discolouration, pain and redness at the implant site. These were generally mild and affected about 1 in 5 people. Afamelanotide is contraindicated for people with reduced liver or kidney function. For full details of adverse reactions and contraindications, see afamelanotide's summary of product characteristics.

3.3 Afamelanotide has not been launched in the UK, but the company has stated that the cost of an implant will be £12,020 (excluding VAT).