Clinuvel gets orphan status for Scenesse in HHD

Clinuvel Pharmaceuticals (ASX:CUV) has been awarded orphan drug designation from the European Medicines Agency for Scenesse in the rare skin disorder Hailey-Hailey Disease (HHD).

Clinuvel is currently trialling Scenesse in HHD through a phase II trial in Italy, based on evidence that alpha-melanocyte stimulating hormone has protective and antioxidative effects in cutaneous cells. The active ingredient of Scenesse, afamelanotide, is an analogue of this hormone.

Orphan status will entitle Clinuvel to potential fee reductions, protocol and scientific assistance and - most importantly - 10 years of marketing exclusivity if Scenesse is approved.

HHD is an inherited, lifelong disorder characterised by plaque-like lesions, blisters and ulcerations on skin folds.

Clinuvel is also developing Scenesse as a treatment for erythropoietic protoporphyria (EPP), a disorder causing severe light intolerance, and is waiting the completion of an EMA review into the treatment.

In an announcement designed to address questions from shareholders about why the review process has taken so long, chairman Stan McLiesh and CEO Dr Philippe Wolgen said three factors are responsible.

First, no other melanocortin has ever been submitted to European regulatory bodies, they said. Second, this is the first time evaluation of light exposure is needed to be assessed in a submission.

Third, the EMA has to review safety data for afamelanotide for the last 20 years in general, and nine years in EPP.

The current EMA review is expected to be complete between July and October, but further reviews may be needed. EMA approval for Scenesse would trigger Clinuvel’s New Drug Application with the US FDA.

Clinuvel Pharmaceuticals (ASX:CUV) shares were trading unchanged at $1.42 as of around 2 pm on Monday.