Vimseltinib receives FDA approval for the treatment of tenosynovial giant cell tumor

Vimseltinib receives FDA approval for the treatment of tenosynovial giant cell tumor

The US Food and Drug Administration (FDA) has approved vimseltinib (Romvimzatm) 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 exercise trial that led to 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 advancement for patients with TGCT who need better treatment options. TGCT can be a very challenging disease. Although it is not usually life-threatening, it can have a devastating impact on a person's quality of life. Once diagnosed, people have to live with this disease for the rest of their lives, and it can cause a lot of pain and disability. “

William Tap, MD, Chief of Sarcoma Medical Oncology Services, Memorial Sloan Kettering Cancer Center

TGCT, also called pigmented villonodular synovitis (PVNs), is not considered cancer because it does not 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 men.

Vimseltinib benefits more than two-thirds of patients

Dr. Tap previously presented findings from the exercise trial in June 2024 at the American Society of Clinical Oncology Annual Meeting. The research was also published inThe LancetAt the time of his presentation he was the correct author of the newspaper.

This study included 123 patients, 83 of whom received vimseltinib and 40 of whom received placebo. The researchers reported that 67% of patients who received vimseltinib had measurable reductions in tumor volume compared to none in the placebo group.

Additionally, patients in the study who received vimseltinib reported improvement in several key measures, including:

• Bewegungsbereich
• Physische Funktion
• Gelenksteifigkeit
• Schmerz
• Allgemeine Gesundheit

Vimseltinib has fewer side effects than pexidartinib

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

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

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

“We believe vimseltinib is another wonderful option to treat people with TGCT,” says Dr. Tap.

The exercise trial was funded by Deciphera Pharmaceuticals.

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