The Food and Drug Administration (FDA) has approved Scenesse (afamelanotide; Clinuvel) for the treatment of patients with erythropoietic protoporphyria, a rare genetic metabolic disorder in which patients experience severe biochemical reactions following light exposure. 

Erythropoietic protoporphyria is caused by a deficiency of the enzyme ferrochelatase, which leads to an accumulation of protoporphyrin in the body. In patients with erythropoietic protoporphyria, when the skin is exposed to light, it can cause intense pain, erythema, and in rare cases, blistering. 

Scenesse contains afamelanotide, a melanocortin-1 receptor agonist that increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources. The treatment is administered via a subcutaneous implant by a healthcare professional who has been trained in the procedure. 

The approval of Scenesse was based on data from 2 parallel group trials in which patients with erythropoietic protoporphyria were randomized to receive the treatment or a placebo form of the implant. In the first study (N=93), the primary end point was the total number of hours over 180 days spent in direct sunlight between 10am and 6pm on days with no pain. The data showed that treatment with Scenesse resulted in more hours spent in direct sunlight without pain compared with placebo (64 hours vs 41 hours, respectively).

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In the second study (N=74), the primary outcome measure was the total number of hours over 270 days spent outdoors between 10am and 3pm on days with no pain for which “most of the day” was spent in direct sunlight. Findings from the study showed that the median total number of hours was 6 for Scenesse-treated patients compared with 0.75 for those who received placebo.

As for safety, the most common treatment-emergent adverse events included implant site reaction, nausea, oropharyngeal pain, cough, fatigue, skin hyperpigmentation, dizziness, melanocytic nevus, respiratory tract infection, somnolence, non-acute porphyria, and skin irritation.

“Prior to today’s approval, there were no FDA-approved treatments to help erythropoietic protoporphyria patients increase their light exposure,” said Julie Beitz, MD, director of FDA’s Center for Drug Evaluation and Research Office of Drug Evaluation III. “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.”

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