Ezmekly
Opinion
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EMA has issued an opinion on this medicine
mirdametinib
MedicineHumanOpinion
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
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On 22 May 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional1 marketing authorisation for the medicinal product Ezmekly2, intended for the treatment of plexiform neurofibromas (PN) in adults and children from 2 years of age with neurofibromatosis type 1 (NF1). The applicant for this medicinal product is SpringWorks Therapeutics Ireland Limited.
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Ezmekly will be available as 1 mg and 2 mg hard capsules and 1 mg dispersible tablets. The active substance of Ezmekly, mirdametinib, is a selective, non-competitive mitogen-activated protein kinase 1 and 2 (MEK 1/2) inhibitor (ATC code: L01EE05). By inhibiting MEK, mirdametinib blocks the proliferation and survival of tumour cells in which the rapidly accelerated fibrosarcoma (RAF)‑MEK-extracellular related kinase (ERK) pathway is activated.
The benefits of Ezmekly are its ability to provide durable responses (reduction of the volume of plexiform neurofibromas) in adults and children from 2 years of age with NF1-associated symptomatic, inoperable plexiform neurofibromas, as observed in a single arm trial. The most common side effects with Ezmekly in adults include acneiform dermatitis, diarrhoea, nausea, increased blood creatinine phosphokinase, musculoskeletal pain, vomiting, and fatigue. The most common side effects in children include blood creatine phosphokinase, diarrhoea, acneiform dermatitis, musculoskeletal pain, abdominal pain, vomiting, and headache.
The full indication is:
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.
Treatment with Ezmekly should be initiated by physicians experienced in the diagnosis and the treatment of patients with NF1 related tumours.
Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.
1A conditional marketing authorisation is granted to a medicinal product that fulfils an unmet medical need when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required. The marketing authorisation holder is expected to provide comprehensive clinical data at a later stage.
2This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained
Product details
- Name of medicine
- Ezmekly
- Active substance
- mirdametinib
- International non-proprietary name (INN) or common name
- mirdametinib
- Therapeutic area (MeSH)
- Neurofibromatosis 1
- Neurofibroma, Plexiform
- Anatomical therapeutic chemical (ATC) code
- L01EE
- EMA product number
- EMEA/H/C/006460
Conditional approval
This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.
Orphan
This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.
- Marketing authorisation applicant
- Springworks Therapeutics Ireland Limited
- Opinion adopted
- 22/05/2025
- Opinion status
- Positive
News on Ezmekly
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