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Peer-reviewedCC BY-NC-ND 4.0Future Oncology

Plain language summary of the ReNeu study: a study of mirdametinib for adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas

  • Angela Hirbe1
  • Christopher L Moertel2
  • Dusica Babovic-Vuksanovic3
  • Claas Röhl4
  • Kim Bischoff5
  • Kate Kelts6
  • Abraham J Langseth7
  • Michael D Weber7
  • Beverly Oberlander8
  • Adli Wunderlich9
  • Julie Jared10
  • Timothy R Gershon11
11 affiliations
  1. 1Division of Oncology, Washington University, St. Louis, MO, USA
  2. 2Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA
  3. 3Mayo Clinic, Rochester, MN, USA
  4. 4NF Patients United – Global Network of NF Patient Organisations, The Netherlands
  5. 5Neurofibromatosis Network, IL, USA
  6. 6Children’s Tumor Foundation, NY, USA
  7. 7SpringWorks Therapeutics, Inc, a healthcare company of Merck KGaA, Darmstadt, Germany; Stamford, CT, USA
  8. 8Patient and Caregiver Author
  9. 9Patient Author
  10. 10Caregiver Author
  11. 11Department of Pediatrics, Emory University, Atlanta, GA, USA
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Abstract

Plain Language Summary: What is this summary about?

This is a summary of the ReNeu study. The researchers in this study wanted to know if the drug mirdametinib, also known as GOMEKLI® and EZMEKLY®, could help adults and children 2 years and older with Neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (‘Plexiforms’), also known as NF1-PN.

The study was designed to evaluate whether mirdametinib works and is safe in adults and children living with this disease. To see whether mirdametinib worked, the researchers assessed if it could: Reduce the size of plexiforms.

Reduce pain.

Improve health-related quality of life.

This study included 58 adults and 56 children who had NF1 and a plexiform that was causing significant problems, such as pain, problems with mobility, difficulty breathing, or disfigurement. The participants’ plexiforms also could not be removed with surgery.

What were the ReNeu study results?

In the ReNeu study, mirdametinib helped some adults and children to have: Decreases in the size of their target plexiform.

Reduced pain intensity, including a reduced impact of their pain on daily activities.

Improved health-related quality of life.

During this study, 98% of the adults (57 out of 58) and 95% of the children (53 out of 56) had side effects that the doctors considered to be related to mirdametinib. Most of these side effects were not serious.

What do these study results mean?

Mirdametinib helped to shrink plexiforms, reduce the intensity of the participants’ pain and how much the pain interfered with their daily life, and improve health-related quality of life in adults and children.

The approval of mirdametinib by the FDA in the United States and by the EC in the European Union is based on the ReNeu study. These agencies have approved the use of mirdametinib to treat adults and children 2 years of age and older with NF1 who have plexiforms that cause symptoms and cannot be removed or can only be partially removed by surgery.

Clinical trial number: NCT03962543

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Cite

BibTeXRIS
BibTeX
@article{hirbe2026plain,
  title = {{Plain language summary of the ReNeu study: a study of mirdametinib for adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas}},
  author = {Angela Hirbe and Christopher L Moertel and Dusica Babovic-Vuksanovic and Claas Röhl and Kim Bischoff and Kate Kelts and Abraham J Langseth and Michael D Weber and Beverly Oberlander and Adli Wunderlich and Julie Jared and Timothy R Gershon},
  journal = {Future Oncology},
  year = {2026},
  month = oct,
  doi = {10.1080/14796694.2026.2722495},
  url = {https://doi.org/10.1080/14796694.2026.2722495},
  note = {CC BY-NC-ND 4.0},
}
RIS
TY  - JOUR
TI  - Plain language summary of the ReNeu study: a study of mirdametinib for adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas
AU  - Angela Hirbe
AU  - Christopher L Moertel
AU  - Dusica Babovic-Vuksanovic
AU  - Claas Röhl
AU  - Kim Bischoff
AU  - Kate Kelts
AU  - Abraham J Langseth
AU  - Michael D Weber
AU  - Beverly Oberlander
AU  - Adli Wunderlich
AU  - Julie Jared
AU  - Timothy R Gershon
PY  - 2026
DA  - 2026/10/29
JO  - Future Oncology
DO  - 10.1080/14796694.2026.2722495
UR  - https://doi.org/10.1080/14796694.2026.2722495
AB  - Plain Language Summary: What is this summary about?  This is a summary of the ReNeu study. The researchers in this study wanted to know if the drug mirdametinib, also known as GOMEKLI® and EZMEKLY®, could help adults and children 2 years and older with Neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (‘Plexiforms’), also known as NF1-PN.  The study was designed to evaluate whether mirdametinib works and is safe in adults and children living with this disease. To see whether mirdametinib worked, the researchers assessed if it could: Reduce the size of plexiforms.  Reduce pain.  Improve health-related quality of life.  This study included 58 adults and 56 children who had NF1 and a plexiform that was causing significant problems, such as pain, problems with mobility, difficulty breathing, or disfigurement. The participants’ plexiforms also could not be removed with surgery.  What were the ReNeu study results?  In the ReNeu study, mirdametinib helped some adults and children to have: Decreases in the size of their target plexiform.  Reduced pain intensity, including a reduced impact of their pain on daily activities.  Improved health-related quality of life.  During this study, 98% of the adults (57 out of 58) and 95% of the children (53 out of 56) had side effects that the doctors considered to be related to mirdametinib. Most of these side effects were not serious.  What do these study results mean?  Mirdametinib helped to shrink plexiforms, reduce the intensity of the participants’ pain and how much the pain interfered with their daily life, and improve health-related quality of life in adults and children.  The approval of mirdametinib by the FDA in the United States and by the EC in the European Union is based on the ReNeu study. These agencies have approved the use of mirdametinib to treat adults and children 2 years of age and older with NF1 who have plexiforms that cause symptoms and cannot be removed or can only be partially removed by surgery.  Clinical trial number: NCT03962543
SN  - 1479-6694
SN  - 1744-8301
ER  -