On February 14, 2025, the Food and Drug Administration sanctioned vimseltinib (Romvimza, Deciphera Pharmaceuticals, LLC), a kinase inhibitor, for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) where surgical resection may exacerbate functional limitations or lead to significant morbidity.
Effectiveness and Safety
The efficacy was assessed in MOTION (NCT05059262), a double-blind, multicenter, randomized (2:1), placebo-controlled trial involving patients with TGCT for whom surgical resection could exacerbate functional limitations or result in significant morbidity. Eligible patients possessed a verified diagnosis of TGCT with quantifiable disease (RECIST v1.1), including at least one lesion measuring a minimum of 2 cm.
Patients were randomly assigned to receive either a placebo or vimseltinib, provided at a dosage of 30 mg twice weekly for a duration of 24 weeks during the double-blind phase (Part 1). In the open-label phase (Part 2), patients were permitted to continue vimseltinib, while those on placebos had the option to transition to vimseltinib.
Randomization was stratified by tumor location (lower limb against all others) and area (United States versus Non-US). A total of 123 patients were randomized: 83 to the vimseltinib group and 40 to the placebo group in Part 1.
The primary effectiveness outcome measure was the overall response rate (ORR), evaluated through blinded independent radiological evaluation at week 25. The overall response rate (ORR) was 40% (95% CI: 29%, 51%) in the vimseltinib group and 0% (95% CI: 0%, 9%) in the placebo group (p-value <0.0001).
The median duration of response (DOR) was not attained in the vimseltinib cohort. Following a further 6 months of follow-up, 28 responders (85%) exhibited a DOR of at least 6 months, while 19 responders (58%) shown a DOR of at least 9 months.
The primary endpoint was corroborated by statistically significant enhancements in active range of motion, patient-reported physical functioning, and patient-reported pain in the vimseltinib group relative to the placebo group at week 25.
The predominant adverse reactions (≥20%), encompassing laboratory abnormalities, included elevated aspartate aminotransferase, periorbital edema, fatigue, rash, elevated cholesterol, peripheral edema, facial edema, reduced neutrophils, diminished leukocytes, pruritus, and elevated alanine aminotransferase.
The advised dosage of vimseltinib is 30 mg administered orally twice weekly, with a minimum interval of 72 hours between doses.
Susan Hau is a distinguished researcher in the field of cancer cell therapy, with a particular focus on T cell-based approaches and cancer vaccines. Her work spans several innovative treatment modalities, including CAR T-cell therapy, TIL (Tumor-Infiltrating Lymphocyte) therapy, and NK (Natural Killer) cell therapy.
Hau's expertise lies in cancer cell biology, where she has made significant contributions to understanding the complex interactions between immune cells and tumors.
Her research aims to enhance the efficacy of immunotherapies by manipulating the tumor microenvironment and exploring novel ways to activate and direct immune responses against cancer cells.
Throughout her career, Hau has collaborated with leading professors and researchers in the field of cancer treatment, both in the United States and China.
These international experiences have broadened her perspective and contributed to her innovative approach to cancer therapy development.
Hau's work is particularly focused on addressing the challenges of treating advanced and metastatic cancers. She has been involved in clinical trials evaluating the safety and efficacy of various immunotherapy approaches, including the promising Gamma Delta T cell therapy.
