Merck rare tumour drug Ezmekly backed for NHS use
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Merck KGaA's neurofibromatosis type 1 therapy, Ezmekly, has been recommended by NICE for routine NHS use in England, becoming an important option for children and adults with inoperable plexiform neurofibromas.
Merck KGaA's recently approved neurofibromatosis type 1 (NF1) therapy Ezmekly has been cleared for routine NHS use in England, becoming an option for children as young as two with the rare disease.
Reimbursement authority NICE has given the go-ahead for the use of oral MEK inhibitor Ezmekly (mirdametinib) to treat symptomatic, inoperable plexiform neurofibromas (PN) associated with NF1 in patients aged two to 17, nine months after its approval by the MHRA last December for both children and adults.
NF1 is a genetic condition that causes tumours to develop on the covering of nerve cells, leading to disfigurement and other debilitating complications like pain, motor dysfunction, and malignant peripheral nerve sheath cancers. It affects around 25,000 people in the UK, of whom around 30% to 50% develop PN.
Ezmekly was the first drug to be approved for children as young as two with the genetic disorder in the UK, and the second for NF1 after AstraZeneca/MSD's MEK1/2 inhibitor Koselugo (selumetinib), which was recommended by NICE in 2022 for use in NF1 patients aged three and over.
NICE's final draft guidance (PDF) recommends that clinicians prescribe the least expensive option for paediatric NF1 patients over three years. The list price for Ezmekly is £4,670.80 for a pack of 42 capsules, while for Koselugo it is £4,223.59 for a 10 mg 60-capsule pack and £10,560.00 for 25 mg, although both drugs are being provided to the NHS at a confidential discount.
