Australia’s Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) has announced that its drug SCENESSE® (afamelanotide 16mg) has received its first approval from the European Medicines Agency (EMA) to treat a disease caused by exposure to sunlight. The EMA’s Committee for Medicinal Products for Human Use (CHMP) voted in favour of marketing authorisation for SCENESSE® under exceptional circumstances for the rare genetic disorder erythropoietic protoporphyria (EPP).
SCENESSE® is the first treatment ever to have been developed for EPP, a debilitating disorder which is clinically regarded as extreme intolerance to light and UV (phototoxicity). These patients suffer from intolerable pain, swelling, scarring and a state of distress which may result in hospitalisation.
The first of five trials of SCENESSE® for EPP started in 2006, with Clinuvel pioneering the development of a new formulation of the drug, and designing and executing an entirely novel clinical program for EPP. SCENESSE® is the first drug developed by an Australian company and taken from novel molecule to marketing authorisation for a previously untreated disease.
“Today’s EMA outcome is a landmark achievement by Clinuvel and all those who worked with this program for nearly a decade,” Clinuvel’s Acting Chief Scientific Officer, Dr Dennis Wright said. “My first thoughts go out to the EPP patients and families who have been asking us for the release of the drug for many years.
“The life of a patient with EPP is one of social deprivation, punctuated by periods of second degree burns and unspeakable pain following exposure to daylight or bright indoor lighting. For a decade now my team remained motivated by the unique opportunity to provide a product that could be life altering for these patients,” Dr Wright said.
An estimated 10,000 EPP patients are affected worldwide, 45% of whom live in Europe. In September 2014, the EMA announced that SCENESSE® was to be the first drug to be evaluated as part of a new and progressive European regulatory pilot program integrating patients’ and physicians’ clinical experiences with SCENESSE® in the CHMP’s decision process.
The EC approval acknowledges the challenges of conducting clinical trials in rare and severe disorders and grants access to treatments for patients without alternative treatment.1 The European marketing authorisation covers 31 states.2 Clinuvel and the EMA have agreed to a comprehensive post-authorisation pharmacovigilance plan to follow up EPP patients long term.
“Medical innovation requires an exceptional focus with a consistent strategy, passionate team and long term trust from patients and expert physicians worldwide. It is a privilege to have written pharmaceutical history for these patients. The EMA’s executive management is to be commended for its progressive vision and reform,” Clinuvel’s CEO, Dr Philippe Wolgen said.
“The immediate goal now is to distribute the drug to Europe in the coming months, while we owe it to our American and Asia-Pacific patients to accelerate the regulatory submission and approval of SCENESSE®.”
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