FDA Approves Clinuvel’s Scenesse for Extreme Light Sensitivity


The U.S. Food and Drug Administration (FDA) approved Melbourne, Australia-based Clinuvel’s Scenesse (afamelanotide) for the treatment of erythropoietic protoporphyria (EPP). Company stocks soared 57% at the news.

EPP is a rare genetic disease resulting in light intolerance, forcing patients to live indoors or only come out at night. It is caused by a defect in heme biosynthesis, causing EPP patients to accumulate and store protoporphyrin IX (PPIX) in blood and tissues. Visible light and near-visible ultraviolet radiation photoactivates PPIX, which damages surrounding tissue and causes intolerable pain. It affects one in 75,000 to 200,000 people worldwide. There are no approved treatments for the disease in the U.S.

The drug was approved in adults in Europe in December 2014. It received Orphan Drug Designation in 2008 from the FDA, and eventually received Fast Track Status 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, Clinuvel’s chief scientific officer. “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.”

He added, “The outcome today is greatest for the patients and their families who kept asking us to continue the R&D of Scenesse despite the obstacles we faced. The approval of Scenesse today is deserved and based on its safety and medical benefits to patients.”

Scenesse is an under-the-skin implant. The drug is the company’s only approved product. In 2018 it brought in $17.4 million (U.S.).

The company plans to distribute the drug directly to U.S. hospitals within the next 12 months. It also intends to sell it at the same price globally.

A single price-point “is quite alien to our industry,” company chief executive officer Philippe Wolgen told Bloomberg. “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.”

Scenesse’s New Drug Application (NDA) approval was made under a 505(b)(1) process that integrated safety and befits reports, manufacturing processes and adequate labeling in addition to Clinuvel’s post-marketing proposals to clinically follow-up EPP patients long-term. As part of the review process, FDA reviewed real-world evidence (RWE) from the European distribution of the drug.

In a statement, Wolgen said, “This event is transformational in that we are now accelerating our exchange with the FDA and European Medicines Agency to expand the use of Scenesse in additional indications, as the passing of time has unveiled that our breakthrough technology deserves wider application in modern medicine. I am confident that our teams will understand going forward that art and skills will be required to execute against due dates while containing the costs. If the past had been arduous, the hard labor is just about to start.”

The regulatory process began with clinical trials in Europe in 2006. A Phase II trial in the U.S. started in 2010 followed by a 2013 Phase III trial, which finished that year. In January 2016, the FDA requested the full data sets of the clinical trials, then organized a Workshop on EPP in Silver Spring and invited 150 patients and families to share their experiences living with the disease.

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