Two EU orphan designations for Clinuvel drug

Australia's Clinuvel Pharmaceuticals says that its photo-protective drug CUV1647 has been granted two Orphan Medicinal Product designations by the European Medicines Agency (EMEA).

The first is for erythropoietic porphyria, a rare genetic disease with severe skin symptoms with currently no effective preventative therapy other than avoidance of sun and light. The second is congenital erythropoietic porphyrias, which is even more rarely seen. The orphan drug committee included the disease because the etiology of CEP is similar to EPP and treatment with CUV1647 was thought to be potentially useful to prevent severe skin blistering and wound formation. CUV1647 will be a "first in class" drug offering preventative treatment for phototoxic reactions for both groups of patients, unable to expose themselves to light and UV.

Subject to successful completion of the current Phase III EPP trial due in 2009, Clinuvel will seek EMEA marketing authorization for CUV1647 for the first indication. This will be the final regulatory step before the start of sales in the European Union.