- Media release -
The first new pharmaceutical drug invented and developed for an untreated disorder by any Australian company has received regulatory approval from the European Medicines Agency (EMA). SCENESSE® (afamelanotide 16mg) treats the debilitating genetic disorder porphyria (EPP), or absolute intolerance to sunlight. The fear of lifelong burns requires EPP patients to lead an indoor, isolated existence avoiding any form of light.
SCENESSE® is the first treatment ever to have been developed for EPP.
EPP is characterised by an extreme risk of severe burns (phototoxicity) of the skin resulting in intolerable pain, swelling, scarring and a state of distress. A severe reaction – triggered by exposure to light, particularly sunlight – may result in hospitalisation.
Since 2006 Australian company Clinuvel Pharmaceuticals has invented, developed and tested SCENESSE®. The drug – the first approved from a new family of drugs known as melanocortins – acts as an anti-oxidant and activates melanin in skin, providing patients with a biological barrier between their skin and the various wavelengths of light triggering phototoxic reactions. Clinical trials in 350 adult EPP patients have shown the drug to be well tolerated, to reduce the incidence and severity of reactions, and to dramatically improve patients’ quality of life.
“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,” Clinuvel’s Acting Chief Scientific Officer, Dr Dennis Wright said. “For a decade now my team remained motivated by the unique opportunity to provide a product that could be life altering for these patients.”
The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended that SCENESSE® be approved under exceptional circumstances (EC) after announcing in September that SCENESSE® was the first drug in a European pilot program incorporating patients’ and physicians’ clinical experiences in the regulatory decision process.
The EC approval acknowledges the challenges of conducting clinical trials in rare and severe disorders and enables access to treatments for patients without alternative treatment.1 An EC authorisation will enable Clinuvel to market SCENESSE® for EPP across the 31 EMA states.2 As part of the EC approval, Clinuvel and the EMA have agreed to a comprehensive post-authorisation pharmacovigilance plan to follow up 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 privileged to have written pharmaceutical history for these patients,” Clinuvel’s CEO, Dr Philippe Wolgen said.
“The immediate goal now is to facilitate access for European patients in the coming months, while we owe it to our American patients to accelerate the submission of the SCENESSE® dossier to the US FDA.”
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1 According to Article 14(8) of Regulation (EC) No 726/2004. Further details of Approval under Exceptional Circumstances can be found on the EMA’s website at http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2009/10/WC500004883.pdf
2 The 28 EU member nations as well as Iceland, Liechtenstein and Norway.
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Clinuvel Pharmaceuticals Ltd (Australia)
+61 3 9660 4900
About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) is a global biopharmaceutical company focused on developing drugs for the treatment of a range of severe skin disorders. With its unique expertise in understanding the interaction of light and human skin, the company has identified patient populations with a clinical need for photoprotection and another population with a need for repigmentation. These patient groups range in size from 5,000 to 45 million. Clinuvel’s lead compound, SCENESSE® (afamelanotide 16mg), a first-in-class drug targeting erythropoietic protoporphyria (EPP), has completed Phase II and III trials in the US and Europe,andhasbeenrecommended for marketing authorisation under exceptional circumstances by the European Medicines Agency. Based in Melbourne, Australia, Clinuvel has operations in Europe, the US and Singapore.
For more information go to http://www.clinuvel.com.
Clinuvel is an Australian biopharmaceutical company focussed on developing its photoprotective drug, SCENESSE® (afamelanotide) for a range of UV-related skin disorders resulting from exposure of the skin to harmful UV radiation. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following:
- actual results may and often will differ materially from these forward-looking statements;
- no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for SCENESSE® can or will be achieved;
- no assurances can be given by Clinuvel that, even if its development programme for SCENESSE® is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place
Level 14 / 190 Queen Street Melbourne, Victoria 3000 T +61 3 9660 4900 www.clinuvel.com