NICE published final draft guidance on 3 September 2026 recommending mirdametinib (Ezmekly®) for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas in children and young people aged 2–17 years who require systemic therapy.1 Mirdametinib is a MEK inhibitor indicated where tumours are not amenable to surgery. The eligible population in England is approximately 200 patients per year.
The appraisal is proceeding under GID reference gid-ta11819. A final TA number had not been assigned at the time this brief was compiled.
Blueteq comment: NF1-associated plexiform neurofibromas is a rare paediatric condition with a small but clearly defined eligible population and specific eligibility criteria — the type of drug where a structured prior-approval form is most valuable. If mirdametinib is recommended alongside selumetinib (Koselugo) for the same indication, commissioners will need a form that captures which MEK inhibitor is being prescribed and whether switching criteria have been met.
References
1. Mirdametinib for treating NF1-associated plexiform neurofibromas in children and young people (guidance in development, GID-TA11819). NICE. https://www.nice.org.uk/guidance/indevelopment/gid-ta11819
