The first new pharmaceutical drug invented and developed for an untreated disorder by any Australian company has received regulatory approval from the European Medicines Agency (EMA). Scenesee (afamelanotide 16mg) treats the debilitating genetic disorder porphyria (EPP), or absolute intolerance to sunlight. The fear of lifelong burns requires EPP patients to lead an indoor, isolated existence avoiding any form of light. 

Scenesse is the first treatment ever to have been developed for EPP.

EPP is characterised by an extreme risk of severe burns (phototoxicity) of the skin resulting in intolerable pain, swelling, scarring and a state of distress. A severe reaction – triggered by exposure to light, particularly sunlight – may result in hospitalisation. 

Since 2006 Australian company Clinuvel Pharmaceuticals has invented, developed and tested Scenessse. The drug – the first approved from a new family of drugs known as melanocortins – acts as an anti-oxidant and activates melanin in 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 dramatically improve patients’ quality of life.

For more details, go to: http://www.clinuvel.com/en/investors/news-publications/announcements/2014-announcements/europe-approves-novel-australian-drug-developed-for-rare-sun-disease