Gomekli (mirdametinib) has become the first FDA-approved treatment for both adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN)—tumors that often cannot be surgically removed. While similar medications, such as selumetinib, are only approved in children, Gomekli’s approval marks a significant advancement for people of all ages living with this challenging condition.
How Gomekli Works
According to WebMD.com, NF1 patients may develop plexiform neurofibromas—tumors that grow around nerves and other vital tissues, making surgery risky or impossible. Gomekli works by blocking the action of MEK1 and MEK2 enzymes, which send growth signals to these tumors. By interfering with these signals, Gomekli slows tumor growth and can shrink existing tumors, offering hope where surgery cannot.
How Gomekli Is Taken
Gomekli is taken orally twice daily, about 12 hours apart, in 28-day cycles: 21 days on the drug, 7 days off. The dose is tailored to each person’s height and weight. For patients who have trouble swallowing capsules, grape-flavored tablets can be dissolved in water to create a liquid dose.
Clinical Evidence: The ReNeu Study
The safety and effectiveness of Gomekli were evaluated in the ReNeu study, which included 111 people aged 2 and older with NF1 and inoperable, symptomatic PN. Participants took Gomekli in repeated 28-day cycles, with doses based on body size. MRI scans monitored tumor sizes throughout the study.
The results were promising: 41% of adults and 52% of children had at least a 20% reduction in tumor size, while over half of both groups saw tumor shrinkage of 50% or more. Improvements could be seen as early as four months, with the median time to first tumor reduction about eight months. Many experienced durable responses: 88% of adults and 90% of children maintained their response for at least 12 months.
Impact on Symptoms and Quality of Life
Beyond shrinking tumors, Gomekli improved pain and quality of life. By the fifth treatment cycle, over half of adults and nearly 60% of children reported significant pain relief. These benefits were sustained and even increased by later cycles, with most participants reporting a better overall sense of well-being.
Side Effects and Safety
Common side effects in adults include rash, nausea, vomiting, diarrhea, muscle or joint pain, and fatigue. Children may also experience headaches, abdominal pain, and skin problems near the nails. Serious risks include potential eye, heart, and skin issues—regular monitoring is essential. Gomekli can harm a fetus, so effective birth control is needed during and after treatment.
Other Considerations
Most people in studies used Gomekli for at least a year, but long-term side effects are still being studied. Cost assistance is available for eligible patients through the manufacturer’s support program.
Conclusion
Gomekli offers new hope for people with NF1 and inoperable plexiform neurofibromas, providing a non-surgical treatment option that can shrink tumors, ease pain, and improve daily living. As with any medication, close monitoring and guidance from healthcare providers are essential to maximize benefits and safety.
