Monday, March 30, 2020

Monday, March 30, 2020

FDA grants marketing approval for Australian product that treats rare skin disease

The U.S. Food and Drug Administration (FDA) recently granted marketing approval to use Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP).

Scenesse, a product of Australian drug manufacturer Clinuvel Pharmaceuticals Ltd., increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources. It is an implant that is administered subcutaneously.

"For patients who are suffering from erythropoietic protoporphyria, a rare disorder, exposure to light may be extremely painful," Julie Beitz, M.D., director of FDA's Center for Drug Evaluation and Research Office of Drug Evaluation III, said in a press release. "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."

EPP is caused by mutations leading to impaired activity of ferrochelatase, an enzyme involved in heme production. Heme is a vital component in hemoglobin. 

The decrease in ferrochelatase activity results in an accumulation of protoporphyrin IX (PPIX) in the body. Light reaching the skin can react with PPIX which triggers skin pain, redness and thickening.

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U.S. Food and Drug Administration (FDA)

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