FDA approves vimseltinib for tenosynovial giant cell tumor

The U.S. Food and Drug Administration (FDA) has approved vimseltinib (Romvimza) for adult patients with a rare condition called tenosynovial giant cell tumor (TGCT). TGCT is a tumor of the tissue that lines the joints.

Sarcoma oncologist William Tap, MD, Chief of the Sarcoma Medical Oncology Service at Memorial Sloan Kettering Cancer Center (MSK), led the international phase 3 MOTION trial that resulted in the drug’s approval. Vimseltinib is a type of targeted therapy called a kinase inhibitor and is taken as a pill.

“This approval is an exciting advance for patients with TGCT, who need better treatment options,” Dr. Tap says. “TGCT can be a very challenging disease. Although it is usually not life-threatening, it can have devastating effects on someone’s quality of life. After being diagnosed, people must live with this disease the rest of their lives, and it can cause a lot of pain and disability.”

TGCT, which is also called pigmented villonodular synovitis (PVNS), is not considered a cancer because it doesn’t spread to other parts of the body. Some people with the disease require multiple surgeries, even amputations. It is usually diagnosed in people in their 20s and 30s and is more common in women than in men.

Vimseltinib Benefits More Than Two-Thirds of Patients

Dr. Tap previously presented findings from the MOTION trial in June 2024 at the annual meeting of the American Society of Clinical Oncology. The research was also published in The Lancet at the time of his presentation, and he was co-corresponding author of the paper.

That study included 123 patients, 83 of whom got vimseltinib and 40 of whom got a placebo. The investigators reported that 67% of patients receiving vimseltinib had measurably reduced tumor volume, compared with none in the placebo group.

Additionally, patients in the trial who received vimseltinib reported improvement in several important measures, including:

• Range of motion
• Physical function
• Joint stiffness
• Pain
• Overall health

Vimseltinib Has Fewer Side Effects than Pexidartinib

Dr. Tap previously led clinical trials for another drug for TGCT, called pexidartinib (Turalio®). That drug received approval from the FDA in 2019. But because it can cause liver damage in some patients, investigators sought additional options.

This trial found that vimseltinib had far fewer side effects than pexidartinib. The most common ones were swelling, rash, headaches, and feeling tired, but none were severe.

Importantly, the drug did not appear to cause liver injury in any patients who received it. This is significant because patients need to stay on these medicines long-term to keep their tumors under control.

“We believe that vimseltinib is another wonderful option to consider in treating people with TGCT,” Dr. Tap says.