Australia's Clinuvel Pharmaceuticals has filed Scenesse with regulators in Europe for a rare disease where patients suffer pain and scarring from exposure to sunlight.
Specifically, the firm has submitted a marketing authorisation application for Scenesse (afamelanotide) to the European Medicines Agency as a treatment in adults with erythropoietic protoporphyria (EPP), a disease which causes absolute intolerance of patients’ skin to light. The filing is based on Phase II and III studies of the peptide which activates eumelanin, (the dark pigment) in skin, whereby EPP patients to expose themselves to sunlight without incurring burns.
EPP is a rare genetic disease found mainly in fair-skinned people characterised by severe phototoxicity, which can result in patients leading "an indoors and sheltered life, avoiding light and UV exposure to prevent symptoms", Clinuvel says. There is no approved treatment for EPP, which affects 10,000 people globally, including 4,000 in Europe.
Chief scientific officer Hank Agersborg said “we are confident that we have provided sufficient data to demonstrate that Scenesse is a safe and clinically meaningful treatment for EPP”. He added that “we firmly believe that our application will withstand the rigour of the regulatory review".
Philippe Wolgen, chief executive of the Melbourne-based firm, said the submission is "an important milestone in the evolution of Clinuvel", noting that afamelanotide is the first ever melanocortin filed for marketing approval.
Afamelanotide is a fairly controversial treatment which has in the past been developed for cosmetic use as a tanning agent. However, Dr Wolgen has regularly spoken out against such usage and stated that taking the cosmetic pathway is not a strategy Clinuvel will adopt.