The U.S. Food and Drug Administration (FDA) has approved vimseltinib for adult patients with symptomatic tenosynovial giant cell tumor (TGCT).
TGCT is a rare disease in which the tissue lining the joints and tendons in the body grows abnormally. It is characterized by a noncancerous mass or tumor. There are two types: the local or nodular form (involving tumors along tendons that support the joint, or in one area of the joint) and the diffuse form (involving the entire lining of the joint). Symptoms may include: pain, limitation of movement, and locking of the joint. In some cases, the normal joint structure can be destroyed. The knee is the most commonly affected joint, though it can occur in the hip, shoulder, elbow, ankle, wrist, and rarely the jaw. The cause of TGCT is unknown.
Approval
On February 14, the FDA approved vimseltinib for the treatment of this condition. Vimseltinib is an oral, switch control tyrosinase kinase inhibitor. The approval follows positive data from the MOTION clinical trial. This study was a double-blind, multicenter, randomized, placebo-controlled trial in adult patients with TGCT.
A total of 123 patients were enrolled in part one of this trial. A primary outcome was overall response rate, which was 40% in the vimseltinib arm compared to 0% in the placebo arm. Median duration of response (DOR) was not reached in the vimseltinib arm, however, at an additional six month follow-up, 85% had a DOR of six months or greater and 58% had a DOR of nine months or greater. The primary outcome was supported by statistically significant improvements in active range of motion, patient-reported physical functioning, and patient-reported pain in the vimseltinib arm versus placebo.
The most common adverse events were increased aspartate aminotransferase, periorbital edema, fatigue, rash, increased cholesterol, peripheral edema, face edema, decreased neutrophils, decreased leukocytes, pruritus, and increased alanine aminotransferase.
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To learn more about TGCT and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/