S13 Ep41: The European Approval of Mirdametinib Expands the Treatment Paradigm for NF1-Associated Plexiform Neurofibromas: With Ignacio Blanco, MD, PhD

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.