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The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today published advice on four new medicines.

Afamelanotide (Scenesse) will be available for patients with erythropoietic protoporphyria (EPP) through the ultra-orphan pathway, which supports access to medicines for extremely rare conditions. In patients with EPP, exposure to strong light can cause painful burns, swelling and scars which may leave patients incapacitated, affecting their work and family life. There are currently limited treatment options and afamelanotide may provide an opportunity for patients to spend more time outdoors and to take part in work and social activities. It will be available through NHSScotland for three years while the pharmaceutical company gathers further data on the effects of the medicine. Following this, SMC will review the evidence and make a decision on routine availability in NHSScotland. Further information on the ultra-orphan approach can be found in Scottish Government guidance.

Ravulizumab (Ultomiris) was accepted for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). PNH is a rare and potentially life-threatening blood condition in which red blood cells break apart prematurely. Ravulizumab was considered through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines for end of life and rare conditions. PACE participants highlighted that patients can experience symptoms including severe fatigue, abdominal pain, difficulty swallowing, erectile dysfunction, and life-threatening blood clots. For many, the severe fatigue is extremely debilitating and impacts on daily family, work and social life as well as relationships. Current treatment is by intravenous infusion every two weeks. Ravulizumab would reduce the need for infusions to every eight weeks, reducing the frequency of healthcare visits and relieving some of the psychological burden of living with the condition for patients and their families.

Ozanimod (Zeposia) was accepted for the treatment of relapsing-remitting multiple sclerosis (RRMS), where the patient has flare-ups (relapses) followed by periods with milder or no symptoms (remission). It is used in patients with active disease, which means that patients have relapses or signs of active inflammation on scans. Ozanimod offers another treatment option for patients in a convenient oral form.

The committee also accepted upadacitinib (Rinvoq) for the treatment of severe rheumatoid arthritis that cannot be controlled well enough with disease-modifying anti-rheumatic medicines or if the patient cannot take these medicines. Upadacitinib provides another treatment option for patients with this condition.

SMC chairman Mark MacGregor said:

“The committee is pleased to be able to accept these medicines for use by NHSScotland.”

”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.”

“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.”

More details of all medicines can be found on our website.

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