Mirdametinib Offers Second Targeted Therapy Option Against Rare Genetic Disorder
The FDA has approved a new MEK inhibitor for certain patients with symptomatic neurofibromatosis type 1.
The U.S. Food and Drug Administration (FDA) has approved mirdametinib (Gomekli) for the treatment of patients with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas but are unable to undergo full resection. The approval covers patients 2 years and older.
Mirdametinib is a targeted therapy that can inhibit the signaling of MEK proteins linked to cell growth as well as the development of plexiform neurofibromas and other tumors. This is the first FDA approval for mirdametinib, which follows selumetinib as the second FDA-approved MEK inhibitor for patients with NF1.
The approval was based on results from the multicenter, single-arm phase II ReNeu clinical trial involving adult and pediatric patients 2 years of age and older with symptomatic, inoperable neurofibromas causing significant morbidity.
Among the 114 NF1 patients treated with mirdametinib, 41% of the 58 adults and 52% of the 56 pediatric patients experienced a treatment response, meaning their tumors decreased by at least 20% in volume.
The recommended dose of mirdametinib is 2 mg/m2 of body surface area, twice daily for the first 21 days of each four-week cycle, until disease progression or unacceptable toxicity. The maximum dose is 4 mg twice daily.
NF1 is a genetic disorder in which a patient’s cells produce a faulty version of the neurofibromin protein that normally regulates cell growth. About half of those with NF1 develop plexiform neurofibromas that originate in cells that cover nerves. While typically benign, these unpredictable growths can disrupt local tissue and cause severe symptoms depending on where they occur, and in some cases progress into malignant peripheral nerve sheath tumors. According to federal statistics, NF1 is estimated to affect 1 out of every 3,000 to 4,000 people globally.
The FDA rendered its decision on February 11, 2025. Check this resource for updated information on all therapeutics regulated by the FDA.
