Loading Curie Briefs...
Getting the latest healthcare news for you
Getting the latest healthcare news for you
UK's NICE has approved mirdametinib (Ezmekly) for children as young as 2 with inoperable NF1-linked tumours — the first non-surgical drug option for this age group. Trial data showed 52% of kids achieved meaningful tumour shrinkage, with pain and quality-of-life improvements that held up over time. Around 200 patients are expected to benefit under NHS coverage.
The UK's National Institute for Health and Care Excellence (NICE) has recommended mirdametinib (Ezmekly) for children aged 2–17 with symptomatic, inoperable plexiform neurofibromas tied to neurofibromatosis type 1 (NF1) — a rare genetic condition that causes nerve tissue tumours. This is a landmark moment: it's the first time children as young as 2 have had access to a non-surgical drug treatment for the condition. The only other approved option, selumetinib, is limited to ages 3 and up.
The approval was backed by the phase 2b ReNeu trial — the largest multicentre study of NF1-associated plexiform neurofibromas to date. Kids on mirdametinib saw real, lasting results, with tumour shrinkage and meaningful improvements in pain and quality of life emerging early and holding steady throughout treatment.
By the Numbers:
Why it matters: For families dealing with NF1, surgical options are often off the table. Having a second effective drug — now available to even younger children — expands a critically limited treatment landscape and gives clinicians more flexibility to tailor care.
