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.
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.

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Medicine details

Medicine name:
afamelanotide (Scenesse)

for prevention of photoxicity in adult patients with erythropoietic protoporphyria (EPP). Scenesse is to be administered under a Post-Authorisation Safety Study (PASS) protocol in line with the approved European Medicines Agency Risk Management Plan.

Pharmaceutical company
Clinuvel (UK) Ltd
BNF chapter
Nutrition and blood
Submission type
Not recommended
Date advice published
08 February 2021
Additional notes

Information on our approach to assessing medicines for extremely rare conditions is available on our ultra-orphan webpage and in the Scottish Government’s guidance.