New treatment recommended for inoperable plexiform neurofibromas in children

Getty/AndreyPopov

By Sarah Lowden

A new treatment for inoperable plexiform neurofibromas associated with neurofibromatosis type 1 (NF1) in children and young people has been recommended for use on the NHS.

The National Institute for Health and Care Excellence has recommended that mirdametinib, which is made by Merck Serono and sold under the brand name Ezmekly, be used for tumours caused by NF1.

The tumours, which grow on nerves, are non-cancerous but can be difficult or impossible to surgically remove. They can cause pain, weakness, problems with movement and sight loss.

In its final draft guidance, NICE said around 200 patients up to the age of 17 could benefit from using the treatment.

The only other drug treatment for these tumours, selumetinib, was recommended by NICE in 2022 for children aged three and over.

This new treatment, taken twice a day as a tablet or liquid, has been approved for use in children aged two and above.