Clinuvel Files European Marketing Authorisation Application for SCENESSE® (afamelanotide)
08:50 06.02.12
European regulators to evaluate new drugs safety and efficacy for orphan disease erythropoietic protoporphyria (EPP)
MELBOURNE, Australia & BAAR, Switzerland--(BUSINESS WIRE)--
Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that it has submitted a marketing authorisation application (MAA) for its first-in-class drug SCENESSE® (afamelanotide 16mg implant) to the European Medicines Agency (EMA). The MAA covers the use of SCENESSE® as a prophylactic treatment in adult patients with erythropoietic protoporphyria (EPP), a rare disease which causes absolute intolerance of patients skin to light.
SCENESSE®, which received an orphan drug designation for EPP in 2008, will be reviewed under the EMAs Centralised Procedure. An approval under this scheme will allow Clinuvel to market SCENESSE® in all 27 European Union member states as well as Norway, Iceland and Liechtenstein.
We are confident that we have provided sufficient data to demonstrate that SCENESSE® is a safe and clinically meaningful treatment for EPP, Clinuvels Chief Scientific Officer, Dr Hank Agersborg said. We firmly believe that our application will withstand the rigor of the regulatory review.
This filing is an important milestone in the evolution of Clinuvel, and is another landmark among the innovative therapies developed by the biotech sector, as afamelanotide is the first ever melanocortin filed for marketing approval, Clinuvels CEO, Dr Philippe Wolgen said. In the coming months we will continue our constructive dialogue with the regulatory authorities in order to bring this much-needed therapy to the EPP community across Europe.
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.
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.
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