Clinuvel, FDA agree in principle to Phase III study with SCENESSE® (afamelanotide)
SCENESSE® (afamelanotide 16mg) to be trialled for rare disease in US Phase IIIMelbourne, Australia and Baar, Switzerland, March 15 201
Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that it had reached an in principle agreement with the US Food and Drug Administration (FDA) to conduct a Phase III study of the novel drug SCENESSE® (afamelanotide 16mg implant) in the rare disease erythropoietic protoporphyria (EPP). Clinuvel is currently working to finalise the Phase III study (CUV039) protocol with the FDA following an End-of-Phase-II meeting held on Monday March 12.
Study Design
After the completion of pivotal EPP studies in 2011 (CUV029 and CUV030), the expectation is that CUV039 will follow a near-identical design. This Phase III trial will complete the EPP program. Pending final comments by the FDA, it is expected that this study will start in May 2012.
"We are delighted that the FDA have confirmed that our intention to treat EPP has merit, since the Division recognised that this is a severe, debilitating disorder which currently lacks an effective therapy," Clinuvel's Chief Scientific Officer, Dr Hank Agersborg said. "The FDA stated that it will expedite the use and availability of afamelanotide for EPP patients pending final results of this study. The input from the FDA during this meeting was very constructive and encouraging for our program and patients."
Erythropoietic protoporphyria (EPP)
EPP is a rare genetic disease found mainly in fair-skinned people. It is 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. Symptoms can vary from mild to extreme lasting pain requiring hospitalisation. Patients often lead an indoors and sheltered life, avoiding light and UV exposure to prevent symptoms. Presently there is no known effective treatment for EPP, which affects approximately 10,000 people globally, an estimated 4,000 in Europe.
During the Phase II and III studies in Europe, the US and Australia, SCENESSE® has been shown to enable EPP patients to expose themselves to (sun)light without incurring characteristic burns (phototoxicity). Pivotal trials (CUV029 and CUV030) showed SCENESSE® could reduce the severity of EPP symptoms and enable patients to lead more normal lives. A marked improvement in Quality of Life was also reported. Thus far no serious safety concerns have been identified from the use of afamelanotide in more than 650 patients involved in trials, including more than 250 EPP patients. SCENESSE® is being proposed as the first effective treatment to prevent phototoxicity in EPP and was designated as an orphan drug by the FDA in 2008.
Afamelanotide 16mg controlled-release formulation (SCENESSE®)
Afamelanotide, the active ingredient in SCENESSE®, is a linear peptide which activates eumelanin, the dark pigment, in skin. Eumelanin protects skin from light and UV radiation (photoprotection). SCENESSE® is administered underneath the skin as a dissolvable implant, approximately the size of a grain of rice, which activates eumelanin for a period of two months. In February 2012 Clinuvel filed a marketing authorisation application with the European Medicines Agency for EPP. |
