On February 11, 2025, the FDA approved mirdametinib (Gomekli), a kinase inhibitor, for patients aged 2 years and older with symptomatic neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN) that are inoperable.

PN are benign nerve sheath tumours commonly seen in NF1 and their size and prevalence increase with age. Many are inoperable, often causing significant pain and other debilitating symptoms. This approval addresses the critical unmet need for alternatives to pain management and surgery in these patients^.1

The approval was based on the ReNeu trial (NCT03962543), a single-arm study involving 114 patients (58 adults, 56 paediatric). Participants received 2 mg/m² BID in 3-week on/1-week off cycles. 

The study met its primary endpoint, demonstrating confirmed overall response rates (ORR) of 41% in adults and 52% in paediatric patients (P<.001).

The median treatment duration was 22 months, showing significant reductions in PN volume and improvements in pain severity, pain interference, and health-related quality of life (HRQoL).

The treatment was well tolerated, with reported common side effects including rash, diarrhoea, nausea, musculoskeletal pain, vomiting, and fatigue in adults, and abdominal pain, headache, and left ventricular dysfunction in paediatric patients. 


Watch a Summary of the Trial