following a full submission assessed under the orphan medicine process:
selumetinib (Koselugo®) is not recommended for use within NHSScotland.
Indication Under Review: as monotherapy for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above.
In an open-label, single-arm phase II study in paediatric patients with NF1 and symptomatic inoperable PN, selumetinib was associated with a response rate of 66%.
The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Download detailed advice854KB (PDF)
Medicine details
- Medicine name:
- selumetinib (Koselugo)
- SMC ID:
- SMC2540
- Indication:
As monotherapy for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above.
- Pharmaceutical company
- Alexion Pharma UK Ltd
- BNF chapter
- Central nervous system
- Submission type
- Full
- Status
- Not recommended
- Date advice published
- 07 August 2023