Clinuvel Pharmaceuticals (ASX: CUV) will soon begin a Phase III study of its novel drug SCENESSE® in the U.S. to treat the rare disease erythropoietic protoporphyria (EPP), a disease that results in a severe intolerance to light.
The company is working with the U.S. Food and Drug Administration (FDA) to finalise the Phase III study (CUV039) protocol.
EPP is a rare genetic disease found mainly in fair-skinned people, characterised by severe phototoxicity (intolerance to light) of the skin resulting in intolerable pain, swelling and scarring, usually of exposed areas such as the face, hands and feet.
Currently there is no known effective treatment for EPP, which affects about 10,000 people globally.
During Phase II and III studies in Europe, the U.S. and Australia, SCENESSE® has been shown to enable EPP patients to expose themselves to sunlight without incurring characteristic burns.
Pivotal trials showed the drug could reduce the severity of EPP symptoms and enable patients to lead more normal lives.
SCENESSE® is administered underneath the skin as a dissolvable implant, about the size of a grain of rice.
The drug activates eumelanin, the dark pigment in the skin that protects skin from light and UV radiation, for a period of two months.
SCENESSE® is being proposed as the first effective treatment to prevent phototoxicity in EPP and has been designated orphan drug status by the FDA.
The FDA has indicated it will expedite the use and availability of SCENESSE® for EPP patients, pending final results of the study.
The study, which concludes the EPP program, is expected to begin in May.
Last month, Clinuvel filed a marketing authorisation application with the European Medicines Agency for EPP.
If approved, the marketing authorisation would allow the company to market SCENESSE® across the European Union.