Overview

Approved Indications

GOMEKLI™ is indicated for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

Mechanism of Action (MoA)

Mirdametinib is a drug that inhibits mitogen-activated protein kinase 1/2 (MEK1/2), which are key regulators of the extracellular signal-regulated kinase (ERK) signaling pathway. In laboratory tests, it effectively blocked MEK1/2 activity and reduced ERK phosphorylation. In a mouse model of NF1, oral administration of mirdametinib led to decreased ERK phosphorylation, tumor volume, and cell proliferation in neurofibromas.

Dosing & Administration 

Capsules (1 mg and 2 mg) and Tablets for Oral Suspension (1 mg)

The recommended dosage of GOMEKLI™ is 2 mg/m2 orally twice daily, with or without food, for the first 21 days of each 28-day cycle. Treatment is continued with GOMEKLI™ until disease progression or unacceptable toxicity.

Place in Therapy

Neurofibromatosis type 1 is a genetic disorder affecting around 100,000 individuals in the United States. Patients with NF1 face a 30-50% lifetime risk of developing PNs, tumors that grow along peripheral nerves, causing pain, disfigurement, and functional impairments. Approximately 40,000 people in the U.S. live with NF1-related PNs, many of whom are adults with no approved treatment options until the introduction of GOMEKLI™. Plexiform neurofibromas' can also progress into malignant peripheral nerve sheath tumors, which are aggressive and potentially fatal. Surgical removal is often difficult due to the tumors’ infiltrative growth along nerves, with up to 85% of cases deemed inoperable. GOMEKLI™ is the first and only FDA-approved treatment for both adults and children aged 2 years and older with symptomatic PNs not amenable to complete resection. GOMEKLI™ can cause side effects and carries significant safety warnings for ocular toxicity, left ventricular dysfunction, dermatologic adverse reactions, and embryo-fetal toxicity. 

Expected Cost

GOMEKLI™ has a Commercial Copay Program that allows eligible patients with commercial insurance to pay as little as $0 for a 21-day supply of GOMEKLI™. Additionally, the Patient Assistance Program provides free medication to uninsured or underinsured patients, or those without coverage for GOMEKLI™.