Ezmekly
What Ezmekly is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Conditional approval: approved on less complete data because the need is high; more data must follow.
Key facts
- Brand
- Ezmekly
- Active substance
- mirdametinib
- Disease area
- Neurofibromatosis 1; Neurofibroma, Plexiform
- Medicine group
- Antineoplastic agents
- ATC code
- L01EE
- Marketing authorisation holder
- Merck Europe B.V.
- Authorised in the EU since
- 2025
Official information
In the USA
Same active substance: Gomekli
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Ezmekly used for?
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.
What is the active substance in Ezmekly?
Ezmekly contains mirdametinib.
Who makes Ezmekly?
The EU marketing authorisation holder is Merck Europe B.V..
Is Ezmekly an orphan medicine?
Yes. Ezmekly is designated an orphan medicine in the EU, for a rare disease (Neurofibromatosis 1; Neurofibroma, Plexiform).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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