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Eye Treatment a First for the FDA

Scenesse is new treatment for people with rare, painful light sensitivity disease

Patients suffering from a condition that can make exposure to light be extremely painful known as erythropoietic protoporphyria, now have a new treatment. The U.S. Food and Drug Administration (FDA) this week approved Clinuvel Pharmaceutical's Scenesse (afamelanotide) for erythropoietic protoporphyria (EEP). It is the first FDA-approved treatment for EEP, according to Julie Beitz, MD, director of the FDA’s Office of Drug Evaluation III.

EEP is a rare genic disease caused by mutations leading to impaired activity of ferrochelatase, an enzyme involved in heme production, according to the FDA. A drop in ferrochelatase leads to an accumulation of protoporphyrin IX (PPIX) in blood and tissues. Visible light and near-visible ultraviolet radiation reaching the skin photoactivates PPIX causing intense skin pain and changes. EEP affects one in 75,000 to 200,000 people worldwide.

Scenesse has been available in to adults in Europe since December 2014.The FDA gave it Fast Track Statue in May 2017 and Priority Review in January 2019.

“I cannot start to describe what it means to dedicate a large portion of one’s professional life to a single molecule and for one group of patients, while not knowing the regulatory outcome,” said Dennis Wright, chief scientific officer for Clinuvel, a Melbourne, Australia, company. “However, we kept our awareness of the regulatory risks with a first-in-class melanocortin agonist as we faced the subsequent regulatory challenges. The team kept going.”

The stock price of the company increased by 60% after the FDA approval, and some press reports said that increase added $800 million to Clinuvel's market value.

Scenesse, the company’s only approved product, is an under-the-skin implanted. The company plans to distribute the drug directly to U.S. hospitals within the next year. Philippe Wolgen told Bloomberg that Scenesse will sell at the same price point globally.

“We wanted to show the American payers that they would not be paying more for a pharmaceutical product than the Europeans and the Swiss do,” he said.

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Sources: FDA and Biospace, October 8, 2019

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