Feb. 12, 2025 – The FDA has approved a new pill for a rare genetic disorder called neurofibromatosis type 1 (NF1). It is for adults and children ages 2 and older who have nerve tumors (plexiform neurofibromas) that cause symptoms and cannot be fully removed with surgery.
NF1 is a nervous system disorder that affects about 100,000 people in the United States. It causes tumors to grow on nerves, leading to skin changes, bone problems, and learning difficulties. NF1 can shorten life by eight to 15 years, and 30% to 40% of children with NF1 may get fast-growing tumors called plexiform neurofibromas. These tumors can cause pain, disability, and serious complications, and are often hard to remove with surgery.
The drug, called mirdametinib but sold under the brand name Gomekli, works by blocking enzymes that help tumors grow, which helps shrink them and slow their spread. It’s the first treatment approved for both adults and children with NF1-related tumors.
The FDA’s decision was based on positive results from a clinical trial involving 114 people (58 adults and 56 children) with NF1-related nerve tumors. Within two to six months, 41% of adults and 52% of children saw their tumors shrink by at least 20%. Most people who responded to Gomekli stayed better for at least a year, and many continued to improve over two years, according to a news release by SpringWorks Therapeutics, the drug’s maker.
Common side effects include a rash, diarrhea, body pain, tiredness, nausea, and vomiting. Children may also have headaches, stomach pain, nail infections, and heart issues.
The FDA warned that Gomekli may cause serious eye problems, including blurred vision and retinal damage. Patients should report any vision changes or eye pain to their doctor right away. Patients must inform their doctors if they have heart disease or if they are getting treatment for any heart problems. The drugmaker also warned that Gomekli may harm an unborn baby. Women should take a pregnancy test before starting treatment and use birth control during treatment and for six weeks after the last dose (three months for men).
