This addresses symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1), starting from two years of age.
Ezmekly is now the only therapy approved within the EU to treat both adult and child patients suffering from NF1-PN.
Ezmekly is offered in 1mg and 2mg capsules, as well as a 1mg dispersible tablet.
Gomekli (mirdametinib), a mitogen-activated protein kinase kinase inhibitor (MEK inhibitor), has been approved by the US Food and Drug Administration (FDA) for similar use cases. In the EU, mirdametinib is marketed under the brand name Ezmekly.
The EC’s decision was informed by data from the ongoing Phase IIb ReNeu trial, a multi-centre study involving 114 patients aged two years or older with NF1-PN.
The primary endpoint was met with an objective response rate (ORR) of 41% in adults and an even higher rate of 52% in children. These results were obtained through blinded independent central review.
Additionally, substantial improvements were noted concerning pain relief and quality of life enhancements based on various patient-reported outcome measures.
Moreover, Ezmekly demonstrated a manageable safety profile throughout its clinical evaluation.
NF1 is a genetic disorder characterised by infiltrative tumours that grow along the peripheral nerve sheath, leading to severe disfigurement, pain, and functional impairment.
Plexiform neurofibromas can develop into malignant peripheral nerve sheath tumours, which are aggressive and potentially fatal.
Surgical removal is difficult due to their infiltrative nature, with about 85% deemed inoperable for complete resection.