Ezmekly Cleared by NICE for Youngest NF1 Patients

Merck's Ezmekly, a rare tumour drug for NF1, has been cleared by NICE for routine use in England. This means children as young as two with the condition can now access the treatment.

Ezmekly, also known as mirdametinib, is an oral MEK inhibitor that targets symptomatic plexiform neurofibromas (PN) associated with NF1. NICE's final draft guidance recommends its use in patients aged two to 17.

NF1 is a genetic condition that causes tumours to develop on nerve cells, leading to disfigurement and debilitating complications like pain and motor dysfunction.

The list price for Ezmekly is £4,670.80 per pack of 42 capsules, but the NHS will receive it at a confidential discount.