Clinuvel Pharmaceuticals scores US orphan-drug designation

THE ROADHOUSE PHARMACY: Clinuvel Pharmaceuticals (ASX: CUV) has announced that afamelanotide, the active ingredient in the company’s proprietary drug SCENESSE®, has received orphan-drug designation from the US Food and Drug Administration (FDA) for the rare Hailey-Hailey Disease (HHD, also known as familial benign chronic pemphigus).

Clinuvel exlpained that orphan-drug designation is granted by the FDA’s Office of Orphan Products Development to drugs which have the potential to diagnose or treat rare conditions, which are defined as those affecting less than 200,000 individuals in the United
States.

The company said the orphan designation entitles Clinuvel to technical assistance throughout the development process for HHD, potential fee reductions and tax credits, and seven years’ market exclusivity if approved for marketing by the FDA.

The announcement followed another by Clinuvel that SCENESSE® had received an orphan designation from the European Medicines Agency.

“This designation is the second from a major regulatory agency to recognise the potential of afamelanotide to treat HHD patients, who currently lack an effective therapy,” Clinuvel Pharmaceuticals acting chief scientific officer Dr Dennis Wright said in the company’s announcement to the Australian Securities Exchange.

“We are encouraged by the regulatory support to date and now await the outcomes of the Phase II study.”

SCENESSE® is currently being evaluated as a therapy for HHD in a Phase II study in Italy, following on from earlier findings that the drug has protective and anti-oxidative effects on cutaneous cells.

An earlier two patient observational study in HHD in 2012 gave first evidence that the drug was well tolerated and of clinical benefit.

Email: investorrelations@clinuvel.com

Website: www.clinuvel.com