The European Approval of Mirdametinib Expands the Treatment Paradigm for NF1-Associated Plexiform Neurofibromas: With Ignacio Blanco, MD, PhD

Welcome to OncLive On Air®! I’m your host today, Jax DiEugenio.

In today’s episode, we had the pleasure of speaking with Ignacio Blanco, MD, PhD, about the European Commission’s July 2025 conditional marketing authorization of mirdametinib (Ezmekly) for the treatment of adult and pediatric patients at least 2 years of age with symptomatic, inoperable plexiform neurofibromas (PNs) in the context of neurofibromatosis type 1 (NF1). Dr Blanco is chairman of the National Reference Center for Adult Patients with Neurofibromatosis at the Hospital Universitari Germans Trias i Pujol in Barcelona, Spain.

In our exclusive interview, Dr Blanco discussed the significance of this regulatory milestone for a population that has historically lacked effective medical options. He reviewed the pivotal phase 2b ReNeu clinial trial (NCT03962543)that supported this decision, which enrolled both adult and pediatric patients with nonresectable PNs and evaluated clinical and radiologic response rates. Dr Blanco detailed the study’s findings, including overall response rates of 41% and 52% in the adult and pediatric populations, respectively; meaningful reductions in tumor volume; improvements in patient-reported pain and quality of life; and the favorable safety profile characterized primarily by dermatologic and gastrointestinal adverse effects.

He also shared practical guidance for oncologists about identifying appropriate candidates for treatment, emphasizing the agent's role in patients with symptomatic, inoperable disease and underscoring surgery as the preferred option for operable cases. Finally, Dr Blanco reflected on the broader implications of this approval, including the potential for imatinib to be explored in other NF1-associated tumor manifestations, such as low-grade glioma and cutaneous neurofibromas, with several clinical trials currently underway.

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