Scenesse recommended for rare sun disease in Europe
pharmafile | October 27, 2014 | News story | Sales and Marketing |Â Â CHMP, Clinuvel Pharmaceuticals, EMA, EU, scenesseÂ
The European Medicines Agency is recommending that Scenesse (afamelanotide) be approved for the prevention of severe sun burn in adults with erythropoietic protoporphyria (EPP), a rare genetic disease which causes intolerance to light.
Scenesse, manufactured by Australian biopharma firm Clinuvel Pharmaceuticals, is the first medicine for patients with this condition. A positive recommendation has been given by the Committee for Medicinal Products for Human Use (CHMP), the drug safety and efficacy arm of the EMA.
It will now be passed onto the European Commission, which typically takes around three months to make a final approval.
But the CHMP has also recommended that Clinuvel puts in place a ‘robust risk management plan’ that ensures close surveillance of the safety and efficacy of the medicine. As part of this plan, the company will need to establish a registry of patients to collect safety and efficacy data, the regulator says in a statement.
The drug is from a new family of medicines known as melanocortins. It 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.
In recent clinical trials conducted in 350 adult EPP patients, data showed the treatment to be well tolerated, reducing the incidence and severity of reactions and also improved patients’ quality of life.
In a first for the CHMP, a number of EPP patients were also involved in discussions about the drug and were invited to share their experience of living with this condition.
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. The company told Pharmafile that around 400 patients in the UK are thought to have the condition.
One of those is 42-year-old Lorraine Valentine, who says she ‘negotiates’ looking after her two boys and two girls ‘around darkness’, and ‘shadow hopping’ – which is a term EPP sufferers use to describe the practice of hopping between shadows for refuge.
Valentine says she faces also being struck down by attacks in supermarkets with the reflections from the white walls and flooring.
James Rawnsley, also a Briton with EPP, says that whilst he was on Scenesse during clinical trials it was ‘immediately clear’ that he had received the drug and not the placebo.
When out with his wife going for a scan, he says spent six hours exposed to sunlight without even realising – something he has never been able to do in his lifetime – ‘utterly life-changing’.
Ben Adams
Related Content
Rethinking oncology trial endpoints with generalised pairwise comparisons
For decades, oncology trials have been anchored to a familiar set of endpoints. Overall survival …
Sharp invests $100m in US and EU manufacturing and packaging facilities
Sharp Services, a pharmaceutical packaging and sterile manufacturing specialist, has announced investments totalling $100m across …
Sarclisa recommended for EU approval in newly diagnosed multiple myeloma
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Sanofi’s …
