FDA to Review Afamelanotide NDA for Erythropoietic Protoporphyria

A New Drug Application (NDA) for the treatment of erythropoietic protoporphyria (EPP) – a rare blood disorder in which patients experience severe biochemical reactions following light exposure – has been submitted to the Food and Drug Administration (FDA). 

Due to an enzyme deficiency, EPP patients experience burns, ulcers, and phototoxicity when exposed to sun or artificial light. Scenesse (Clinuvel Pharmaceuticals) is a bimonthly, subcutaneous dose of afamelanotide 16mg. The treatment works by activating melanin in the skin, which can shield chemicals in the blood from light. 

Two multicenter, randomized, double-blind, placebo-controlled Phase 3 trials (N=168) compared subcutaneous afamelanotide 16mg vs placebo every 60 days in patients with EPP in the US and Europe.  The primary efficacy endpoint was the number of hours of direct exposure to sunlight without pain. 

The data showed that the duration of pain-free time was longer in the US afamelanotide group vs placebo group after 6 months (69.4 hours vs 40.8 hours; P =.04) and in the European afamelanotide group vs placebo group after 9 months (6.0 hours vs 0.8 hours; P =.005); the incidence of phototoxic reactions was also lower in the European afamelanotide group (77 vs 146, P =.04). In both trials, the use of afamelanotide improved quality of life. 

The FDA previously granted the treatment Fast Track designation in 2016. Scenesse has been authorized in Europe for use in adult patients with EPP since 2014. The Company expects the FDA’s review of the NDA to be completed by 2019.

For more information visit Clinuvel.com.