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NTUK Welcomes Mirdametinib Recommendation

07 September 2026

Further to Friday evening's brief announcement regarding Mirdametinib, reporting that NICE (The National Institute for Health and Care Excellence) has reviewed the evidence submissions and external assessment report and has reached a conclusion to recommend Mirdametinib as an option to treat symptomatic, inoperable plexiform neurofibromas associated with Neurofibromatosis Type 1 in children and young people aged 2 to 17 years:

Stuart Hill, Merck Medical Director United Kingdom & Ireland, said:

“We welcome NICE’s draft guidance for Mirdametinib as an important step towards broadening treatment options for children and young people aged 2 to 17 years living with NF1-associated symptomatic, inoperable plexiform neurofibromas. Mirdametinb is indicated for children from two years of age, for whom treatment options remain limited. The availability of a dispersible tablet formulation also provides an option designed to support administration in younger children and those who may have difficulty swallowing capsules whole. We look forward to continuing to work with NICE and the NHS as the appraisal progresses”

This news was warmly received by Nerve Tumours UK.

With Karen Cockburn, Charity Director at Nerve Tumours UK, adding:

"I totally endorse Stuart Hill's comment above and am delighted to welcome the good news, on behalf of the NF1 Community, that there is another drug available for children, especially for those aged under 3 and those who cannot swallow capsules. We will await the further decision on those aged 17 and over."

Registered Charity Number: 1078790 and SC045051

Nerve Tumours UK has taken reasonable care to ensure that the information contained on this website and in its newsletters, literature is accurate. Nerve Tumours UK cannot accept liability for any errors or omissions, or for information becoming out-of-date. Any information given is not a substitute for getting appropriate medical advice from your own GP or other healthcare professional.

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INFER (International NF Educational Resources) is a series of online educational lectures for medical professionals by leading neurofibromatosis experts. The Masterclasses take place online approximately once a month, each on a different topic, and include real-time interaction between the expert presenter and the participants. The presentations are conducted in English, with real-time audio interpretation available in 6 additional languages: French, German, Italian, Portuguese, Russian, and Spanish. A recording of each INFER masterclass is then be made available online in each language for those who could not attend an event. INFER is an initiative of Children’s Tumor Foundation Europe, supported by an educational grant from AstraZeneca. https://ctfeurope.org/research/masterclasses-in-nf

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