FDA approves first treatment to increase pain-free light exposure in patients with a rare disorder

FDA on Tuesday approved afamelanotide (Scenesse—Clinuvel), a melanocortin-1 receptor agonist, to boost pain-free light exposure in adults with a history of phototoxic reactions from erythropoietic protoporphyria.

FDA on Tuesday approved afamelanotide (Scenesse—Clinuvel), a melanocortin-1 receptor agonist, to boost pain-free light exposure in adults with a history of phototoxic reactions from erythropoietic protoporphyria. This is the first FDA-approved treatment to help patients with the rare condition, which is caused by mutations leading to impaired activity of ferrochelatase, increase their light exposure. Julie Beitz, MD, director of FDA's Center for Drug Evaluation and Research Office of Drug Evaluation III, said: "Today's approval is one example of the FDA's ongoing commitment to encourage industry innovation of therapies to treat rare diseases, and work with drug developers to make promising new therapies available to patients as safely and efficiently as possible." Afamelanotide is an implant that is administered subcutaneously. The most frequently reported adverse effects of afamelanotide are implant site reaction, nausea, oropharyngeal pain, cough, fatigue, skin hyperpigmentation, dizziness, melanocytic nevus, respiratory tract infection, somnolence, non-acute porphyria, and skin irritation. This application received Priority Review and Orphan Drug designations.