The US FDA has approved afamelanotide (Scenesse, Clinuvel) in treating phototoxic reactions (damage to skin) in patients with erythropoietic protoporphyria.
The drug helps increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria.
“For patients who are suffering from erythropoietic protoporphyria, a rare disorder, exposure to light may be extremely painful.” Julie Beitz, MD, director of the FDA’s Center for Drug Evaluation and Research Office of Drug Evaluation III, said in a statement..
Erythropoietic protoporphyria is a rare condition resulting from mutations that limit the activity of the enzyme ferrochelatase, which is involved in heme production. The decrease in ferrochelatase activity causes protoporphyrin IX (PPIX) to accumulate in the body. When light reaches the skin, it can react with PPIX, resulting in severe skin pain and skin changes, including redness and thickening.
Afamelanotide is a melanocortin-1 receptor agonist that increases eumelanin production in the skin regardless of exposure to sunlight or artificial light.
The approval was based on the results from two parallel-group clinical trials in patients with erythropoietic protoporphyria.
Those in the afamelanotide cohort in the first trial had spent 64 hours in direct sunlight, compared to 41hours among those in the placebo group.
In the second clinical trial patients in the afamelanotide group, had spent a median total number of 6 hours compared with 0.75 hours for those in the placebo group.
The FDA also urges patients to use sun protection measures while receiving afamelanotide treatment.The agency gave this application priority review designation and orphan drug designation