NEWS RELEASE

Department of Defense
Defense Health Agency Research and Development-Medical Research and Development Command
Congressionally Directed Medical Research Programs

Released: February 28, 2025

FORT DETRICK, Md.- On February 11, 2025, the FDA approved a new drug, mirdametinib, for adult and pediatric patients living with neurofibromatosis type 1 and symptomatic plexiform neurofibromas, which are tumors found on the tissue covering and protecting nerves.

Through the Neurofibromatosis Research Program, CDMRP funded a phase 2 clinical trial of mirdametinib as a treatment option for neurofibromatosis type 1. Mirdametinib is now approved to use for patients two years and older.

"CDMRP-funded research transforms health care through novel ideas and development of new treatments to be used in the clinic," Col. Mark Hartell, director of CDMRP, said. "Improving the lives of those who are affected by the diseases and conditions is central to the CDMRP's mission."

Neurofibromatosis is a group of disorders that causes tumors to form in the nervous system, including the brain and spinal cord, as well as anywhere in the body, such as the eyes and muscle tissues.

Most neurofibromatosis tumors are benign, but some may turn cancerous. Although neurofibromatosis is often an inherited condition, between 30 and 50 percent of cases are from of the NF1 gene.

In fiscal 2006, the Neurofibromatosis Research Program funded a Clinical Consortium Award, led by Bruce Korf, M.D., Ph.D., and established the Neurofibromatosis Clinical Trials Consortium, aimed to develop and implement innovative therapies for patients of all ages living with neurofibromatosis.

"The consortium consistently manages a portfolio of research leading to impactful clinical trials that, if successful, will transform the health care of all patients living with neurofibromatosis," Theresa Miller, Ph.D., a program manager at CDMRP, said.

Follow-on NFRP funding in fiscal 2011 supported multiple studies under the consortium, including a phase 2 clinical trial evaluating the radiographic response of plexiform neurofibromas to mirdametinib, a MEK inhibitor, which is a drug that blocks a protein in a pathway found to be overactive in NF1-associated cancers.

The research team published their initial findings from the clinical trial in 2021, showing that mirdametinib decreases plexiform neurofibroma size and decreases pain for neurofibromatosis type 1 patients.

This initial phase 2 trial was followed with a multi-center phase 2b trial funded external to CDMRP. The phase 2b trial, called ReNeu, further demonstrated mirdametinib as a promising treatment for adults and children with neurofibromatosis type 1 associated plexiform neurofibromas.

"The consortium is proud to have performed the initial phase 2 study, NF106, led by Brian Weiss, M.D., Chief of the Division of Hematology/Oncology/Stem Cell Transplant at Indiana University, that was the first to show the efficacy of mirdametinib for adolescents and adults with NF1-associated plexiform neurofibroma and spurred the development of the ReNeu study," Michael Fisher, M.D., group chair of the consortium, said.

The NFRP-funded consortium brings together multiple organizations and individuals with a common goal of executing clinical trials to translate new neurofibromatosis treatments, like mirdametinib, to patients.

"We are grateful to the CDMRP Neurofibromatosis Research Program for creating and supporting the NF Clinical Trials Consortium," Fisher said.

For more information about the Congressionally Directed Medical Research Programs, our research programs, and research highlights, please visit the website at https://cdmrp.health.mil/.

Point of Contact:
CDMRP Public Affairs
usarmy.detrick.medcom-cdmrp.mbx.cdmrp-public-affairs@health.mil

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