EU Approves First Neurofibromatosis Drug, Marking a Breakthrough in NF1-PN Treatment
On July 18, 2025, the European Commission (EC) granted conditional marketing authorization for EZMEKLY (mirdametinib) to SpringWorks Therapeutics, Inc. (a Merck & Co., Inc. healthcare company) for the treatment of adult and pediatric patients (2 years and older) with neurofibromatosis type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas (PN).
On July 18, 2025, the European Commission (EC) granted conditional marketing authorization for EZMEKLY (mirdametinib) to SpringWorks Therapeutics, a Merck & Co., Inc. (MSD) subsidiary, for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) associated with neurofibromatosis type 1 (NF1) in patients aged two years and older. As the first and only treatment approved in the EU for both adults and children with NF1-PN, this approval addresses a long-standing unmet medical need for this disease and marks a new milestone for the global neurofibroma treatment industry. The drug was previously approved by the US FDA for the same indication, and this EU approval further validates its clinical value.
EZMEKLY's approval was based on positive results from the ReNeu trial. The trial enrolled 114 patients (58 adults and 56 children) with inoperable NF1-PN. Data showed a confirmed objective response rate (ORR) of 41% in adults and an even higher ORR of 52% in children. The median best reduction in target PN volume was 41% in adults and 42% in children, respectively. Responses lasted for at least 12 months in 88% of adults and 90% of children, with over half experiencing remissions for more than 24 months. Furthermore, patients experienced early and sustained significant improvements in pain symptoms and quality of life, providing strong data support for clinical treatment.
In terms of safety and industry impact, EZMEKLY demonstrated manageable safety and tolerability. Common adverse reactions in adults included acneiform dermatitis and diarrhea, while in children, increased creatine kinase and diarrhea were the primary adverse reactions, all within clinically manageable ranges. This approval not only brings new hope to NF1-PN patients, but also promotes the industry's attention to the research and development of drugs targeting the RAS/MAPK signaling pathway, provides an important reference for the subsequent development of therapies for similar diseases, and helps the field of neuro-oncology treatment move towards precision and full population coverage.