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 name:
- afamelanotide (Scenesse)
- SMC ID:
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
- Date advice published
- 08 February 2021