Merck, a leading science and technology company, announced that the European Commission (EC) has granted conditional marketing authorization for EZMEKLY® (mirdametinib). This approval covers the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in both pediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above. Importantly, EZMEKLY is the first and only therapy approved in the EU for both adults and children with NF1-PN. SpringWorks Therapeutics Inc., a healthcare company under Merck, received the approval.
Breakthrough for NF1-PN Patients
Experts hailed the approval as a significant advancement, particularly for adult patients who previously had no approved treatments. Dr. Ignacio Blanco of Spain noted that EZMEKLY offers a dissolvable tablet, helping patients who struggle to swallow pills. Similarly, the Children’s Tumor Foundation emphasized that this milestone was possible through collaboration between researchers, industry, and patient advocacy groups.
Strong Clinical Results Support Approval
As per the press release, the EC’s decision is based on data from the Phase 2b ReNeu trial, which enrolled 114 patients (58 adults and 56 children). The trial showed objective response rates (ORR) of 41% in adults and 52% in children, with tumor volume reductions averaging over 40%. Notably, most patients sustained their responses for at least 12 to 24 months. Participants also reported early and lasting improvements in pain and quality of life.
Manageable Safety Profile
EZMEKLY demonstrated a manageable safety and tolerability profile. Common side effects included dermatitis acneiform, diarrhea, and elevated blood creatine phosphokinase levels in both adults and children.
Addressing an Urgent Need
NF1 affects roughly 135,000 people in the EU, with up to 50% developing PN, which are often inoperable and can lead to severe complications. With this approval, Merck takes a major step in addressing this unmet medical need across Europe.