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.”

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.pdf2 The 28 EU member nations as well as Iceland, Liechtenstein and Norway.

Media enquiriesJournalista (US/Europe)James Tout, +44 207 232 4671james@journalista.co.ukorClinuvel Pharmaceuticals Ltd (Australia)Lachlan Hay, +61 3 9660 4900Lachlan.Hay@clinuvel.com

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