The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended conditional marketing authorization for mirdametinib, a MEK inhibitor developed by SpringWorks Therapeutics, for treating symptomatic, inoperable plexiform neurofibromas (PN) in NF1 patients aged two and older. A final decision from the European Commission is anticipated in Q3 2025. If approved, mirdametinib would be the first and only authorized therapy for both adults and children with NF1-PN in the European Union.
This potential approval addresses a significant unmet medical need. NF1-PN, affecting an estimated 135,000 individuals in the EU, can lead to severe pain, disfigurement, and functional impairment. Existing surgical options are often limited due to the tumors’ infiltrative growth pattern. Mirdametinib offers a new treatment approach, potentially improving the quality of life for these patients and addressing a debilitating aspect of NF1.
The CHMP recommendation stems from the Phase 2b ReNeu trial, which demonstrated substantial tumor reduction in both adult and pediatric patients. The trial achieved confirmed objective response rates of 41% in adults and 52% in children, with a median reduction in tumor volume of 41% and 42%, respectively. Responses were generally durable, with a large percentage of patients experiencing responses lasting at least 12 months. The safety profile was manageable, with common adverse events including rash, diarrhea, nausea, and musculoskeletal pain.
This positive CHMP opinion marks a crucial step towards providing a much-needed therapy for NF1-PN patients in Europe. Approval could significantly impact the treatment landscape for this rare disease, offering a non-surgical option with demonstrated efficacy and a manageable safety profile. This development also reinforces the value of targeted therapies in addressing complex genetic disorders and highlights the potential for continued advancements in NF1 treatment.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
