The orphan designation is for the preventative treatment of erythropoietic porphyrias (metabolic blood disorders). The designated diseases, erythropoietic porphyrias (EPP), are classified as severe and rare genetic diseases with no effective preventative therapy other than avoiding sun and light, by spending life indoors.
Subject to successful completion of the company’s current Phase III EPP trials due in 2009, Clinuvel will seek marketing authorization from Swissmedic and the registration of CUV1647. This will be the final regulatory step before the start of sales in Europe.
Philippe Wolgen, Clinuvel’s CEO, said: “The acknowledgement by both EMEA and Swissmedic, within two consecutive months, highlights the severity of erythropoietic diseases and recognizes the potential value of CUV1647 as a preventative therapy to such patients. Switzerland will be an important market for Clinuvel.”