Clinuvel updates on EU access

Company News

Clinuvel Pharmaceuticals (ASX:CUV) has updated investors on the company’s post-authorisation distribution of SCENESSE (afamelanotide 16mg) across Europe for adult patients with the rare disorder erythropoietic protoporphyria (EPP).

SCENESSE is the first approved treatment for EPP, a genetic disorder characterised by acute phototoxic reactions (anaphylactoid reactions and burns) and forced withdrawal from exposure to all forms of visible light.

Clinuvel conducted clinical trials of SCENESSE in EPP from 2006 to 2013. During this time the company evaluated the treatment in 352 EPP patients. After completion of the clinical trial program, approximately 130 patients across eight countries (Australia, Austria, France, Germany, Italy, Sweden, Switzerland and The Netherlands) received SCENESSE free of charge.

"In total 601 implant injections were provided to patients free of charge under compassionate use schemes. The development program and the following compassionate use program was supported entirely by equity funding," said the company.

Subsidised access to SCENESSE treatment, prior to the drug’s formal commercial approval, was made possible in both Italy and Switzerland from 2010 and 2012 respectively. These two programs enabled 115 EPP patients to benefit from treatment with SCENESSE totalling more than 200 patient years of cumulative care.

"Clinuvel is no longer in the position to provide access to SCENESSE on a subsidised basis," it said.

The European Medicines Agency (EMA) granted SCENESSE marketing authorisation under a strict risk management plan, comprising a Post-Authorisation Safety Study (PASS) as well as a Retrospective Chart Review (RCR) to follow up those who do not consent to participate in the PASS.

SCENESSE is only being made available to EPP patients through Porphyria Expert Centres, which require training and accreditation by Clinuvel.

According to the company, in Germany it is anticipated that the first year three Porphyria Expert Centres – in Berlin, Chemnitz, and Düsseldorf – will be able to prescribe the treatment.

"The German national authorities have released their first assessment of the use of SCENESSE in EPP. In parallel, 13 German insurance firms have agreed to make SCENESSE available to EPP patients. It is the company’s expectation for a uniform commercial price to be in place across Europe. Discussions are underway with authorities in nine countries to make SCENESSE available, with further submissions anticipated in 2016," said the company.

”The long term success of SCENESSE lies in the safety profile of the product for EPP patients and, hence, our and regulators’ focus on pharmacovigilance,” said Clinuvel’s chair, Mr Stan McLiesh. “Now we look forward to the release of the photoprotective product by the various ethics committees to finally make the wishes of EPP patients a reality.

“After years of subsidising the treatment and providing it free of charge, Clinuvel needs to move towards the next stage of development: investing in a treatment for children with EPP. It has long been our desire to bring light to these children. Both decision makers and the medical community agree that Clinuvel should provide a pharmaceutical answer to EPP children,” added Mr McLiesh.