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European Commission Grants Conditional Approval of EZMEKLY® (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN
Globenewswire·2025-07-18 15:30

Core Insights - The European Commission granted conditional marketing authorization for EZMEKLY (mirdametinib) for treating symptomatic, inoperable plexiform neurofibromas (PN) in patients aged 2 years and older with neurofibromatosis type 1 (NF1), making it the first therapy approved in the EU for both adults and children with NF1-PN [1][11]. Company Overview - SpringWorks Therapeutics, a healthcare company of Merck, focuses on developing treatments for rare tumors and has commercialized the first FDA-approved medicine for adults with desmoid tumors and the first approved medicine for both adults and children with NF1-PN [19]. - The company emphasizes addressing significant unmet needs and improving outcomes for patients and their families [3]. Clinical Trial Insights - The approval of EZMEKLY is based on the Phase 2b ReNeu trial, which enrolled 114 patients (58 adults and 56 pediatric patients) and demonstrated an objective response rate (ORR) of 41% in adults and 52% in children [3][6]. - The trial showed a median best percentage change in target PN volume of -41% in adults and -42% in children, with significant improvements in pain and quality of life reported [3][6]. Treatment Profile - EZMEKLY is available in 1 mg and 2 mg capsules, as well as a 1 mg dispersible tablet that dissolves easily in water, catering to patients who have difficulty swallowing pills [5]. - The treatment has a manageable safety profile, with common adverse reactions including dermatitis acneiform (83% in adults), diarrhea (55%), and nausea (55%) [4]. Disease Context - NF1 affects approximately 3 in 10,000 people in the EU, equating to an estimated 135,000 individuals, with a lifetime risk of developing plexiform neurofibromas ranging from 30% to 50% [2][7]. - Plexiform neurofibromas can lead to severe disfigurement, pain, and functional impairment, and are often not amenable to surgical resection due to their infiltrative growth pattern [8][9].