The FDA has approved vimseltinib, a new oral medication designed to treat tenosynovial giant cell tumor (TGCT), a rare, noncancerous tumor that affects the joints. This approval offers a new treatment option for adults with symptomatic TGCT when surgery is not viable or could worsen their condition.
TGCTs develop due to a mutation in the CSF1 gene, which leads to excessive production of colony-stimulating factor 1 (CSF1) protein. This overproduction results in tumor formation in the joints or surrounding tissues, causing pain, swelling, stiffness, and difficulty in movement. While surgery has been the primary treatment, these tumors frequently recur, and multiple procedures can damage the joints over time. This has created a need for more effective and less invasive treatment alternatives.
Marketed under the brand name Romvimza, the newly approved drug works by blocking enzymes that allow CSF1 protein to fuel tumor growth. By interfering with this process, Romvimza helps shrink the tumors and reduce inflammation, providing relief from symptoms. The medication is an oral capsule that is expected to be commercially available in the United States this week. The FDA has recommended a twice-weekly dosage, with at least 72 hours between doses.
The approval of Romvimza was based on results from a clinical trial involving patients with TGCT who were not candidates for surgery. Participants received either a placebo or 30 milligrams of Romvimza twice a week over a 24-week period. The study found that the drug significantly reduced tumor size in 40% of patients, compared to none in the placebo group. Additionally, patients experienced improvements in movement, function, and pain relief. Among those who responded to the treatment, 85% experienced benefits lasting at least six months, while 58% continued to benefit for more than nine months.
Like many medications, Romvimza is associated with potential side effects. The most common include elevated liver enzyme and cholesterol levels, fatigue, rash, itching, swelling in the legs, face, or around the eyes, and a reduction in white blood cell counts. Since the drug can impact liver function, patients should inform their doctors about any preexisting liver or kidney conditions before beginning treatment.
Patients should also disclose their full medical history, including allergies and any prescription or over-the-counter medications they take, to prevent possible drug interactions. Romvimza may pose risks to unborn babies, so both men and women undergoing treatment are advised to use birth control and continue using it for at least one month after their last dose. Additionally, breastfeeding mothers are advised to refrain from breastfeeding while on the medication and to consult their healthcare providers regarding their options.
The introduction of Romvimza provides new hope for individuals suffering from TGCT, offering a non-surgical alternative that can help manage symptoms and improve quality of life.FDA Approves Romvimza: New Oral Treatment for Rare Joint Tumor
