29. Scenesse

fda
(FDA)

Active ingredient: afamelanotide
Disease: erythropoietic protoporphyria
Peak sales estimate: n/a
Approved: Oct. 9, 2019
Company: Clinuvel

The scoop: Once the target of multiple buyout attempts, Clinuvel opted to go it alone with its sole drug candidate for rare disease erythropoietic protoporphyria (EPP). After years of work, Clinuvel’s patience paid off with the FDA approval of under-the-skin implant Scenesse (afamelanotide) in October. Cleared in Europe in 2014, Scenesse is now the first FDA-approved treatment for EPP, which affects between 75,000 and 200,000 individuals worldwide, Clinuvel said. The company is studying the implant for other uses, including pigmentation disorder vitiligo, and has three other preclinical programs working. In a statement (PDF), Clinuvel CEO Philippe Wolgen, M.D., said the drugmaker intends to push ahead with U.S. and EU regulators to broaden the market for Scenesse, its only approved treatment. Wolgen told Bloomberg the company plans to sell Scenesse directly to U.S. hospitals and clinics—where the implants will be placed—at a price point that matches that in other countries, a move he called “quite alien to our industry.” –– Kyle Blankenship

29. Scenesse

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