The United States Food and Drug Administration (FDA) has approved Scenesse (afamelanotide) intended to increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria, a rare and painful skin disorder caused by mutations leading to impaired activity of ferrochelatase, an enzyme involved in heme production, it was reported yesterday.

The efficacy of the product was established in two parallel group clinical trials in patients with erythropoietic protoporphyria who received Scenesse or a placebo form of the implant subcutaneously every two months. The first clinical trial enrolled 93 patients, of whom 48 received Scenesse, and were followed for 180 days. The second clinical trial enrolled 74 patients, of whom 38 received Scenesse, and were followed for 270 days.

Julie Beitz, MD, director of FDA's Center for Drug Evaluation and Research Office of Drug Evaluation III, said, 'For patients who are suffering from erythropoietic protoporphyria, a rare disorder, exposure to light may be extremely painful. Prior to today's approval, there were no FDA-approved treatments to help erythropoietic protoporphyria patients increase their light exposure. 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.'

The FDA granted this application Priority Review designation. Scenesse also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.