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FDA approves Clinuvel’s Scenesse for rare genetic disorder

Published on 09/10/19 at 08:52am

The US FDA has approved Clinuvel’s Scenesse (afamelanotide 16mg) as the first ever treatment for a rare genetic metabolic disorder which causes absolute intolerance to light.

Scenesse has been developed and approved to “increase pain free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoprophyria (EPP)”.

Dr Dennis Wright, Chief Scientific Officer at Clinuvel, said: “Today is a memorable day and victory for EPP patients, their families, and the global medical community who have all supported the development and US approval of the first ever treatment for this debilitating condition and the world’s first systemic photo protective drug.

“EPP patients are born with the disease and need to avoid all sources of light exposure, including sunlight throughout their life, or risk incapacitating burns. Scenesse is a novel drug and formulation, developed by Clinuvel to provide photo protection for EPP patients, enabling patients to expose to light, providing them a freedom they never had.”

The drug itself binds to the melanocortin-1 receptor on skin cells and sets in motion a cascade of cellular events, one of which is the activation of the pigment melanin to provide a physical barrier to visible and invisible light in EPP patients.

The drug is administered as a 16mg controlled-release injectable implant, designed to provide protection for up to 60 days.

Scenesse was approved by the European Medicines Agency for EPP in 2014 and launched in 2016.

Dr Philippe Wolgen added: “Our team is granted very little time to celebrate and now needs to shift its focus to facilitating drug product access for US EPP patients.”

Nik Kiran

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