The Medicines and Healthcare products Regulatory Agency (MHRA)\u00a0has today (11 December 2025) approved the medicine mirdametinib (Ezmekly) for the treatment of plexiform neurofibromas\u00a0in adults\u00a0and\u00a0adolescents. It\u00a0is the first treatment\u00a0for children as young as 2 years\u00a0old\u00a0with a genetic condition called neurofibromatosis type 1 (NF1).\u00a0<\/p>\n
NF1\u00a0is\u00a0a\u00a0rare genetic condition\u00a0that\u00a0can cause tumours to form on nerves throughout the body.\u00a0These\u00a0tumours, known as\u00a0plexiform neurofibromas,\u00a0may grow and press on nearby tissues, sometimes causing pain or problems with daily\u00a0activities.\u00a0\u00a0<\/p>\n
Mirdametinib\u00a0works by blocking certain signals in the body that would otherwise allow these tumours to grow.\u00a0It\u00a0is available as a dispersible tablet, which\u00a0can be swallowed whole or dissolved in water. This\u00a0makes\u00a0it suitable for patients unable or unwilling to swallow a capsule\u00a0and\u00a0particularly suitable\u00a0for\u00a0children under 6 years of age.\u00a0\u00a0<\/p>\n
Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said:\u00a0\u00a0<\/p>\n
\u201cThe approval of\u00a0mirdametinib\u00a0provides the first\u00a0treatment\u00a0for NF1 that can be used for\u00a0children aged\u00a0as young as\u00a02 years and older.\u00a0Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.\u00a0\u00a0<\/p>\n
\u201cAs with all licensed medicines, we will continue to monitor its safety and effectiveness closely.\u201d\u00a0<\/p>\n
A full list of side effects can be found in the Patient Information Leaflet (PIL) or the Summary of Product Characteristics (SmPC), which will be published on the MHRA website within 7 days of approval.\u00a0<\/p>\n