NICE Approves Mirdametinib for NF1 Patients Aged 2-17 Years
The National Institute for Health and Care Excellence (NICE) has recommended mirdametinib (Ezmekly, Merck Serono) as a treatment option for children and young people aged 2-17 years with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1 (NF1).
This decision expands access to non-surgical drug treatment for this age group, which was previously limited. Mirdametinib is the second targeted therapy available for NF1, alongside selumetinib, and both are MEK inhibitors.
The recommendation was supported by findings from the phase 2b ReNeu trial, which showed that among paediatric participants, 29 of 56 children (52%) achieved a confirmed objective response during the 24-cycle treatment period. This meant a reduction of at least 20% in target tumour volume, with a median best percentage reduction of 42% and reductions of up to 91% reported.
Treatment was also associated with improvements in patient- and parent-reported outcomes, including reductions in worst tumour pain severity and pain interference, alongside improved health-related quality of life. Mirdametinib showed a manageable safety profile in the trial, with nearly all children experiencing treatment-related adverse events.