FDA Greenlights Romvimza for Rare Joint Tumor Treatment

The U.S. Food and Drug Administration (FDA) has approved Romvimza, a new treatment for Tenosynovial Giant Cell Tumors (TGCT), marking a significant advancement for patients with this rare joint condition. On February 18, 2025, the FDA announced its approval based on promising results from a clinical trial involving TGCT patients who could not undergo surgery. The drug, produced by Deciphera Pharmaceuticals, targets adults with symptomatic TGCT where surgical intervention could potentially worsen the patient's condition.

Romvimza works by blocking enzymes that assist the CSF1 protein, which is responsible for tumor growth, thereby reducing both tumor size and inflammation. In the clinical trial, patients received either a placebo or 30 milligrams of Romvimza twice a week for 24 weeks. Results showed that Romvimza shrank tumors in 40% of patients, compared to 0% in the placebo group. Additionally, it improved movement, function, and pain for those affected by TGCT.

The clinical trial revealed that most patients (85%) experienced benefits from Romvimza for approximately six months, with 58% seeing continued benefits for over nine months. The FDA recommends a twice-weekly dosing schedule, ensuring at least 72 hours between doses to maximize efficacy and minimize potential side effects.

While Romvimza offers hope for many, it does come with warnings. The drug can affect liver function; therefore, patients should disclose any existing liver or kidney problems to their healthcare providers before commencing treatment. Moreover, because Romvimza may harm an unborn baby, both men and women are advised to use birth control during treatment and for one month after the last dose.

TGCTs are noncancerous tumors that arise in the joints or surrounding tissues due to a faulty CSF1 gene. Excessive production of the colony-stimulating factor 1 (CSF1) protein leads to tumor growth in TGCTs. The FDA's approval of Romvimza provides a new therapeutic strategy by addressing this underlying cause.

Romvimza, marketed under its brand name, is expected to be commercially available in oral capsule form in the United States this week. The availability of this medication offers new hope for those with limited treatment options, potentially improving quality of life by alleviating symptoms and enhancing mobility.