Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

PURPOSE: To determine whether afamelanotide can reduce the severity of phototoxic reactions in patients with EPP. THEORETICAL FRAMEWORK: EPP is a genetic photosensitivity disorder where the mainstays of management are covering up from sunlight, systemic beta carotene and the use of controlled courses of UVR treatment. One of the mechanisms for the protective effects of UVR treatment is the increase in melanin content of the skin. UVR treatment causes DNA damage to skin cells and increases the risk for skin cancers, hence it is unwise for this to be used on a recurring basis. Afamelanotide, through its ability to stimulate melanin production without causing the DNA damage associated with UVR treatment, appears to be a promising agent to combat this distressing disorder. STUDY DESIGN: This is a phase III, randomised, placebo controlled study to evaluate the safety and efficacy of subcutaneous implants of afamelanotide in patients suffering from EPP. The study will be performed in compliance with Good Clinical Practice (GCP) including the archiving of essential documents. METHODOLOGY: The target population consists of male and female participants. Up to 70 patients with diagnosed EPP (from past case history) and fulfilling the necessary inclusion/exclusion criteria will be enrolled. Potential study patients will be identified from each centre's records of patients with well characterised history (or documented diagnosis) of EPP. Patients will be enrolled and will receive afamelanotide (16 mg implants) or placebo according to the following dosing regime: * Group A will be administered active implants on Days 0, 60, 120, 180 and 240. * Group B will be administered placebo implants on Days 0, 60, 120, 180 and 240.