• The ReNeu trial (NCT03962543) showed mirdametinib reduced the overall response rate by 41% in adults and 52% in pediatric patients with NF1 plexiform neurofibromas not amenable to complete resection (HR not applicable; 95% CI: 29-55 for adults; 95% CI: 38-65 for pediatrics) ^[1].
• The trial enrolled 114 patients, including 58 adults and 56 pediatric patients, aged 2 years and older ^[1].
• Event rates: 41% of adults and 52% of pediatric patients achieved a confirmed overall response ^[1].
• The primary endpoint was the confirmed overall response rate, defined as the percentage of patients with a complete response or partial response (≥20% reduction in PN volume) ^[1].
• Secondary endpoints included safety and tolerability assessments, which indicated common adverse reactions in both adult and pediatric cohorts ^[1].
• Mirdametinib is administered at a dose based on body surface area, taken orally twice daily for 21 days, followed by a 7-day treatment break ^[1].
• Clinicians should consult current prescribing information for full dosing guidance.

What is mirdametinib (Gomekli) approved for?

Mirdametinib (Gomekli) is approved for the treatment of adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas that are not amenable to complete resection, as of February 11, 2025.

How does mirdametinib work?

Mirdametinib is a MEK inhibitor that targets the MAPK/ERK signaling pathway, which is often dysregulated in tumors associated with neurofibromatosis type 1, leading to reduced tumor growth and proliferation.

What is the recommended dose of mirdametinib?

Mirdametinib is administered based on body surface area, taken orally twice daily for 21 days, followed by a 7-day treatment break to complete a 28-day treatment cycle. Clinicians should consult current prescribing information for full dosing guidance. Full dosing guidance is available in the prescribing information for Gomekli.

What are the most common side effects of mirdametinib?

Common side effects of mirdametinib include rash, diarrhea, nausea, musculoskeletal pain, and left ventricular dysfunction, among others.

How does mirdametinib compare to other treatments for NF1?

Mirdametinib represents a targeted therapy option for NF1-associated plexiform neurofibromas, particularly for patients with inoperable tumors, compared to traditional surgical approaches which may not be feasible due to the tumor's location or size.

Who qualifies for mirdametinib treatment?

Patients aged 2 years and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that are not amenable to complete surgical resection qualify for treatment with mirdametinib.