Drug to treat rare sunlight skin-burning condition approved

A drug has been made available through the NHS which could allow people with a rare disease to spend more time outside.

Afamelanotide – Scenesse – will be available for patients with erythropoietic protoporphyria (EPP) through the ultra-orphan pathway, which supports access to medicines for extremely uncommon conditions.

People with the disease can suffer painful burns, swelling and scars due to exposure to strong light – leaving them incapacitated, affecting their work and family life.

The move comes as Scottish Medicines Consortium (SMC) published advice on four new medicines on Monday.

“We know availability of this medicine will be welcomed and we hope it will provide some benefit to patients.”
Mark MacGregor, SMC chairman

There are currently limited treatment options for EPP and it is hoped the drug may provide an opportunity for patients to spend more time outdoors and to take part in activities.

Mark MacGregor, SMC chairman, said: “The committee is pleased to be able to accept these medicines for use by NHS Scotland.

”For those with EPP, afamelanotide may improve their symptoms, enabling them to spend more time outside.

“We know availability of this medicine will be welcomed and we hope it will provide some benefit to patients.”

The drug will be available through NHS Scotland for three years while the pharmaceutical company gathers further data on the effects of the medicine.

SMC will then review the evidence and make a decision on routine availability through the health service.

Ravulizumab – Ultomiris – was accepted for the treatment of paroxysmal nocturnal haemoglobinuria (PNH), a potentially life-threatening condition in which red blood cells break apart prematurely.

Ozanimod – Zeposia – was given the green light for the treatment of relapsing-remitting multiple sclerosis (RRMS), where the patient has flare-ups followed by periods with milder or no symptoms (remission).

The committee also accepted upadacitinib – Rinvoq – for the treatment of severe rheumatoid arthritis that cannot be controlled well enough.

Mr MacGregor added: “In our PACE meeting for ravulizumab, patient groups and clinicians told us how the symptoms of PNH can have a significant impact on patients and their families.

“As ravulizumab requires intravenous infusion on an eight weekly basis, it may allow patients to enjoy a better quality of life with fewer disruptions for treatment.

“For patients with relapsing-remitting MS, ozanimod offers another treatment with the benefit of oral delivery.

“Our decision on upadacitinib provides those with severe rheumatoid arthritis with another treatment option if they have not responded to current treatments.”