Clinuvel has developed Scenesse for the treatment of the genetic disorder EPP
The first new pharmaceutical drug developed for a rare genetic disease that causes intolerance to sunlight has received regulatory approval from the European Medicines Agency (EMA).
Scenesse (afamelanotide 16mg), developed by the Australian company Clinuvel Pharmaceuticals, treats erythropoietic protoporphyria (EPP).
EPP is characterised by an extreme risk of severe burns (phototoxicity) of the skin resulting in intolerable pain, swelling, scarring and distress. A severe reaction – triggered by exposure to light, particularly sunlight – may result in hospitalisation.
Scenesse, the first drug approved from a new family called melanocortins, acts as an anti-oxidant and activates melanin in the skin, providing patients with a biological barrier between their skin and the various wavelengths of light triggering phototoxic reactions.
Clinical trials in 350 adult EPP patients have shown the drug to be well tolerated, to reduce the incidence and severity of reactions, and to improve quality of life.
The Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Scenesse under exceptional circumstances (EC), reflecting the difficulties of conducting placebo-controlled trials, which will enable Clinuvel to market the drug in 31 European countries.
As part of the approval, Clinuvel and the EMA have agreed to a comprehensive post-authorisation pharmacovigilance plan to follow up patients long term.