, which provides oncology professionals with the resources and information they need to provide the best patient care. In both digital and print formats, 

 covers every angle of oncology practice, from new technology to treatment advances to important regulatory decisions.

In today’s episode, we had the pleasure of speaking with Christopher L. Moertel, MD, about the 

(Gomekli) for the treatment of adult and pediatric patients at least 2 years of age with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PNs) that are not amenable to complete resection. Dr Moertel is a pediatric neuro-oncologist, professor, program director of the Pediatric Neuro-Oncology Fellowship Program, a faculty member in the Department of Pediatrics, medical director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs, co-medical director of the Katie Hageboeck Children’s Cancer Research Fund Clinic, the clinical neuro-oncology leader of the Brain Tumor Program, and the Kenneth and Betty Jayne Dahlberg Professor at the University of Minnesota School of Medicine in Minneapolis.

In our exclusive interview, Dr Moertel discussed the significance of this approval, key efficacy and safety data from the phase 2 ReNeu trial (NCT03962543), and considerations for integrating mirdametinib into clinical practice for patients with NF1-associated PN.

That’s all we have for today! Thank you for listening to this episode of 

. Check back on Mondays and Thursdays for exclusive interviews with leading experts in the oncology field.

For more updates in oncology, be sure to visit www.OncLive.com and sign up for our e-newsletters.

 is also on social media. On X and Bluesky, follow us at @OncLive. On Facebook, like us at OncLive, and follow our OncLive page on LinkedIn.

, please consider subscribing to our podcast on Apple Podcasts, Spotify, and many of your other favorite podcast platforms,* so you get a notification every time a new episode is posted. While you are there, please take a moment to rate us!

*OncLive On Air is available on: Apple Podcasts, Google Podcasts, Spotify, Amazon Music, Audacy, CastBox, Deezer, iHeart, JioSaavn, Listen Notes, Player FM, Podcast Addict, Podchaser, RadioPublic, and TuneIn.

Podcasts

Christopher L. Moertel, MD, discusses the significance of the FDA approval of mirdametinib, based on efficacy and safety data from the phase 2 ReNeu trial.

FDA Approval Insights: Mirdametinib for NF1-Associated Plexiform Neurofibromas: With Christopher L. Moertel, MD

6368672538112

“The approval of mirdametinib is significant because this a highly selective MEK inhibitor that will provide benefit to both adults and children with neurofibromatosis type 1 who have plexiform neurofibromas that are not amenable to surgery and are causing significant quality-of-life or pain issues.”

Christopher L. Moertel, MD, pediatric neuro-oncologist; professor; director, Pediatric Neuro-Oncology Fellowship Program, Division of Pediatric Hematology/Oncology; faculty, Department of Pediatrics; medical director, Pediatric Neuro-Oncology and Neurofibromatosis Programs; co-medical director, Katie Hageboeck Children's Cancer Research Fund Clinic; clinical neuro-oncology leader, Brain Tumor Program; and The Kenneth and Betty Jayne Dahlberg Professor, University of Minnesota School of Medicine, discusses the FDA approval of mirdametinib (Gomekli) for the treatment of adult and pediatric patients with neurofibromatosis type 1 (NF1)–associated plexiform neurofibromas (PN).

 for adult and pediatric patients 2 years of age and older with NF1 who have symptomatic PN not amenable to complete resection.

The regulatory decision was supported by data from the phase 2b ReNeu trial (NCT03962543), which demonstrated that treatment with mirdametinib led to significant reductions in tumor volume, pain relief, and improvements in quality of life for patients with NF1-PN that was not amenable to surgical resection. The trial enrolled both adult and pediatric patients and evaluated mirdametinib’s efficacy and safety in this patient population.

Findings that supported the approval showed that adult patients (n = 58) achieved a confirmed overall response rate (ORR) of 41% (95% CI, 29%-55%); pediatric patients (n = 56) experienced a confirmed ORR of 52% (95% CI, 38%-65%).

Beyond tumor reduction, the trial demonstrated significant clinical benefits in symptom management. Patients reported marked reductions in pain severity, which translated into improved daily functioning and quality of life.

Moertel emphasizes that this approval is particularly meaningful for adult patients with NF1-PN, who have historically lacked FDA-approved treatment options.

The approval of mirdametinib represents a significant advancement in the treatment landscape for NF1-PN, Moertel concludes. Future research may focus on optimizing long-term disease management strategies and evaluating combination approaches to further enhance clinical outcomes.

Videos

Christopher L. Moertel, MD, discusses the FDA approval of mirdametinib for patients with neurofibromatosis type 1–associated plexiform neurofibromas.

Christopher L. Moertel, MD

Articles

FDA Approval Insights: Mirdametinib for NF1-Associated Plexiform Neurofibromas: With Christopher L. Moertel, MD

Dr Moertel on the FDA Approval of Mirdametinib for NF1-Associated Plexiform Neurofibromas