Ezmekly is a medicine used to treat plexiform neurofibromas, benign (non-cancerous) tumours that grow along nerves, in adults and children 2 years and older with neurofibromatosis type 1 (NF1). It is used when the tumours cause symptoms (such as pain and weakness) and cannot be removed by surgery. Neurofibromatosis is rare, and Ezmekly was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 25 July 2019. Ezmekly contains the active substance mirdametinib.
Therapeutic Indication
### Therapeutic indication Ezmekly as monotherapy is indicated for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above.
Therapeutic Area (MeSH)
ATC Code
L01EE
ATC Item
Mitogen-activated protein kinase (MEK) inhibitors
Pharmacotherapeutic Group
Antineoplastic agents
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| mirdametinib | N/A | Mirdametinib |
EMA Name
Ezmekly
Medicine Name
Ezmekly
Aliases
N/ANo risk management plan link.
