The National Institute for Health and Care Excellence has rejected Clinuvel UK’s afamelanotide for treating erythropoietic protoporphyria (EPP) in preliminary guidelines deeming it far too expensive for NHS use.

EPP is an inherited genetic disorder affecting around 500-600 people in England, which causes accumulation of chemical substances called porphyrins in the body. The primary symptom is hypersensitivity of the skin to sunlight and some types of artificial light, which leads to an often rapid and painful chemical reaction under the skin (phototoxicity).

There is currently no effective treatment for the underlying cause of EPP, or to protect against or relieve pain caused by phototoxicity.

Afamelanotide (also called Scenesse) is a dissolving implant placed under the skin up to four times per year, which works by stimulating the production of melanin, which contributes to photoprotection primarily by acting as a filter, absorbing UV and visible light.

However, in draft recommendations, the Institute says the true benefit of the treatment remains unclear, and that its cost effectiveness ranged from £278,000 to £1.7 million per QALY gained, and thus far above the £100,000 ceiling for rare disease therapies evaluated as part of NICE’s Highly Specialised Technologies programme.

The appraisal committee agreed that afamelanotide is innovative and has non-health-related benefits, which should be taken into account in its decision-making, but even so it was unlikely that the drug would be considered a cost-effective use of NHS resources.