SpringWorks Therapeutics, a healthcare company of Merck, has secured the European Commission (EC) conditional marketing authorisation for Ezmekly (mirdametinib).
Ezmekly, also known as Gomekli in the US, is an oral, small-molecule MEK inhibitor.
The drug is indicated for symptomatic, inoperable plexiform neurofibromas (PN) in children and adults aged two years and above, with neurofibromatosis type 1 (NF1).
It was approved by the US Food and Drug Administration (FDA) for the same indication.
Also, Ezmekly has been granted Orphan Drug designations from both the US FDA and the EC for the treatment of NF1.
Ezmekly is the first treatment approved in the European Union (EU) for both adults and children with NF1-PN.
Merck rare tumour business global head Jan Kirsten said: “Bringing innovation to patients living with rare tumours around the world is a clear reflection of our focus on addressing significant unmet needs and transforming outcomes for patients and their families.
“With the European approval of Ezmekly, the first therapy approved for both adults and children with NF1-PN, we are taking a major step toward improving care for this underserved community and are committed to making our medicine available to eligible NF1-PN patients across Europe as quickly as possible.”
The EC’s decision is based on data from the ongoing Phase 2b ReNeu trial, a multi-centre, open-label, single-arm study involving 114 NF1-PN patients aged two years or older.
The Phase 2b trial met its primary endpoint, showing an objective response rate (ORR) of 41% in adults and 52% in children, as assessed by blinded independent central review.
The trial also showed a median best percentage change in target PN volume of -41% in adults and -42% in children.
Among those with a confirmed response, 88% of adults and 90% of children had a response lasting at least 12 months, while 50% of adults and 48% of children for least 24 months.
Ezmekly showed significant improvements in pain and quality of life, assessed through multiple patient-reported outcome tools, and a manageable safety and tolerability profile.
Dermatitis acneiform, diarrhoea, and nausea are common adverse reactions in adults, while increased blood creatine phosphokinase and diarrhoea in children.
SpringWorks Therapeutics is offering Ezmekly in 1mg and 2mg capsules, as well as a 1mg dispersible tablet that dissolves easily in water.
Children’s Tumor Foundation (CTF) CEO Annette Bakker and CTF Europe director Dariusz Adamczewski said: “This European Commission approval is an important milestone for NF patients and caregivers, as it means more treatment options for patients with plexiform neurofibromas, including adults.
“This is the kind of progress that happens when researchers, industry and organisations like ours work together with a shared focus on delivering new treatments for patients.”
Recently, Merck completed the acquisition of SpringWorks, a biopharmaceutical company based in Stamford, Connecticut, for $3.4bn.
