afamelanotide (Scenesse)

following a full submission assessed under the ultra-orphan process:

afamelanotide (Scenesse®) is not recommended for use within NHSScotland.

Indication under review: prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP).

In a phase III study, afamelanotide increased the duration of time, over a six-month period, that patients with EPP spent in direct sunlight on pain-free days compared with placebo.

The submitting company’s justification of the treatment’s cost in relation to its benefits was not sufficient and in addition the company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
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From 8 February 2021, afamelanotide (Scenesse) can be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated. After 3 years the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.