扒哥吃瓜

The Medicines and Healthcare products Regulatory Agency (MHRA)聽has today (11 December 2025) approved the medicine mirdametinib (Ezmekly) for the treatment of plexiform neurofibromas聽in adults聽and聽adolescents. It聽is the first treatment聽for children as young as 2 years聽old聽with a genetic condition called neurofibromatosis type 1 (NF1).聽

NF1聽is聽a聽rare genetic condition聽that聽can cause tumours to form on nerves throughout the body.聽These聽tumours, known as聽plexiform neurofibromas,聽may grow and press on nearby tissues, sometimes causing pain or problems with daily聽activities.聽聽

Mirdametinib聽works by blocking certain signals in the body that would otherwise allow these tumours to grow.聽It聽is available as a dispersible tablet, which聽can be swallowed whole or dissolved in water. This聽makes聽it suitable for patients unable or unwilling to swallow a capsule聽and聽particularly suitable聽for聽children under 6 years of age.聽聽

Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said:聽聽

鈥淭he approval of聽mirdametinib聽provides the first聽treatment聽for NF1 that can be used for聽children aged聽as young as聽2 years and older.聽Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.聽聽

鈥淎s with all licensed medicines, we will continue to monitor its safety and effectiveness closely.鈥澛�

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.聽

Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme, either through the website聽() or by searching the Google Play or Apple App stores for MHRA Yellow Card.鈥�

Notes to editors鈥�鈥�聽

• Marketing authorisation approval was granted on 11 December 2025 to SpringWorks Theraputics Ireland Limited.

• At聽the time of approval, only one medicine was authorised in the UK for plexiform neurofibromas in patients aged three years and older with NF1. Mirdametinib provides an additional treatment option and has been shown to be effective in patients from a slightly younger age, starting at聽2聽years聽of age.聽

• More information can be found in the Summary of Product Characteristics and Patient Information Leaflets which will be published on the MHRA Products website within 7 days of approval.聽聽

• The Medicines and Healthcare products Regulatory Agency (MHRA)聽is responsible for聽regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.聽聽

• The MHRA is an executive agency of the Department of Health and Social Care.聽聽

• For media enquiries, please contact newscentre@mhra.gov.uk or call 020 3080 7651.