Mirdametinib Represents Long-Awaited Approved Treatment Option for Adult NF1-Associated PNs

Author: Chris Ryan

Published On: 07/17/25

In the following article Justin T. Jordan, MD, MPH, FAAN, discusses the implications of the FDA approval of mirdametinib for adult NF1-associated plexiform neurofibromas. Full disclosure: Dr. Jordan serves as a board member with the NF Network. Our reprint of this is only educating our community and not promoting the medication or endorsing its use.

The FDA approval of mirdametinib (Gomekli) marked a key milestone for adult patients with neurofibromatosis type 1 (NF1)–associated plexiform neurofibromas (PNs), with an agent now indicated for the treatment of this population for the first time, according to Justin T. Jordan, MD, MPH, FAAN.

In February 2025, the FDA approved mirdametinib for adult and pediatric patients 2 years of age and older with NF1 who have symptomatic PNs not amenable to complete resection. This approval was supported by data from the phase 2 ReNeu trial (NCT03962543), where the MEK inhibitor generated an overall response rate of 41% (95% CI, 29%-55%) in adult patients (n = 58) and 52% (95% CI, 38%-65%) in pediatric patients (n = 56).

This agent represents another tool to add to the comprehensive support network available for this patient population, Jordan added.

NF1 is a genetic disorder that affects not only the patient but also the entire family. When a person is diagnosed with NF1, plugging the patient and family into larger NF1 communities is important, such as support groups and patient advocacy organizations,” Jordan said during an interview with OncLive®. “I can't say enough about how important those organizations are in terms of providing support to patients and families, and in helping spur on research.”

During the interview, Jordan expanded on past treatment challenges for adult patients with NF1-associated PNs, delved into the implications of the FDA approval of mirdametinib, and highlighted key management strategies when utilizing MEK inhibitors in this setting.

Jordan is an associate professor of neurology, head of the Neuro-Oncology Section, and an associate professor at the O'Donnell Brain Institute at UT Southwestern Medical Center in Dallas, Texas.

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