The European Commission (EC) has granted conditional approval to SpringWorks Therapeutics’ Ezmekly (mirdametinib) to treat neurofibromatosis type 1 (NF1), a rare genetic disorder affecting an estimated 135,000 people in the EU.
The Merck KGaA healthcare company’s oral small molecule MEK inhibitor has been authorised to treat adult and paediatric patients aged two years and older who have symptomatic plexiform neurofibromas (PN) that cannot be removed surgically.
Patients with NF1 have a 30% to 50% lifetime risk of developing PNs, tumours that grow along the peripheral nerve sheath. They can cause severe disfigurement, pain and functional impairment, and may also develop into malignant peripheral nerve sheath tumours, an aggressive and potentially fatal disease.
Due to the infiltrative tumour growth pattern of PNs along nerves, surgical removal can be challenging, with up to approximately 85% of PNs considered not amenable to complete resection.
The EC’s decision makes Ezmekly the first and only therapy approved in the EU for both adults and children with NF1-PN and comes five months after the US Food and Drug Administration approved the drug for the same indication under the brand name Gomekli.
Both approvals were supported by results from the phased 2b ReNeu trial, which demonstrated an objective response rate of 41% and 52% in adult and paediatric NF1-PN patients, respectively.
Among those with a confirmed response, 88% of adults and 90% of children were in response for at least 12 months, while 50% of adults and 48% of children were in response for at least 24 months. Patients in both cohorts also experienced early and sustained improvements in pain and quality of life.
Jan Kirsten, global head of Merck Healthcare’s rare tumour business, said: “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.”
Also welcoming the authorisation, Ignacio Blanco, chairman of the National Reference Center for Adult Patients with Neurofibromatosis at Hospital Universitari Germans Trias i Pujol, said: “Patients with NF1-PN often face physical and mental health challenges and impaired quality of life given the limited treatment options available for this lifelong and debilitating disease.
“This approval represents an important advance, especially for adults who previously did not have an approved treatment.”
