Around 200 children and young people with a rare genetic condition that causes tumours to grow on nerves could soon gain access to a new NHS-funded treatment after it was recommended by NICE.
The National Institute for Health and Care Excellence has issued final draft guidance recommending mirdametinib for the treatment of symptomatic plexiform neurofibromas, non-cancerous tumours associated with neurofibromatosis type 1 (NF1).
NF1 is a rare inherited condition that affects the growth of nerve tissue and can lead to tumours developing throughout the body. While these growths are non-cancerous, they can be extremely challenging to remove surgically because of their location and may cause chronic pain, mobility difficulties, muscle weakness, vision problems and significant impacts on quality of life.
The latest recommendation marks an important step forward for families, extending access to drug treatment to children aged just two years old for the first time.
Until now, the only NICE-approved drug treatment for these tumours has been selumetinib, which was recommended in 2022 for children aged three and above. NICE accepted clinical advice that younger children are likely to experience similar benefits from treatment.
Clinical evidence submitted to NICE indicates that mirdametinib can reduce the size of plexiform neurofibromas and may offer outcomes that are at least comparable to, and potentially better than, existing treatment options. Evidence also suggests the therapy could be associated with fewer side effects, although NICE noted that findings are based on indirect comparisons rather than head-to-head clinical trials.
The treatment is administered twice daily, either as a tablet or a liquid formulation, and works by blocking signalling pathways that drive tumour growth.
NICE approved the medicine through its cost comparison process, concluding there is sufficient evidence that it delivers patient benefit while representing value for money for routine NHS use.
Under the guidance, clinicians will work with children and their parents or carers to determine the most appropriate treatment option. Decisions will take into account factors including administration methods, dosing requirements and overall cost, with the least expensive suitable treatment expected to be selected.
If the guidance is formally published, NHS England will be required to fund mirdametinib within 30 days where it is considered the most appropriate treatment choice.
The medicine is manufactured by Merck Serono and marketed under the brand name Ezmekly.
For NHS leaders and specialist paediatric services, the recommendation represents another important development in improving access to targeted therapies for rare diseases, while expanding treatment options for families affected by NF1.
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