Surgical resection remains the most common treatment approach for people living with tenosynovial giant cell tumors (TGCT). Unfortunately, surgery is not always an appropriate treatment option for all people. As TGCT Support, a Patient Worthy partner, shares, there are a few other treatment options that are, in some cases, effective for TGCT. However, this is not always the case. As a result, it is incredibly important to identify novel therapeutics that could transform the lives of those living with tenosynovial giant cell tumors.
Deciphera Pharmaceuticals’ investigational therapy vimseltinib is being explored as a potential treatment option for TGCT. According to reporting from Drugs.com, Deciphera Pharmaceuticals recently released positive topline data from the Phase 3 MOTION trial. Within this trial, researchers evaluated vimseltinib for people with TGCT for whom surgery is not an option.
What’s the Deal with Vimseltinib?
Deciphera Pharmaceuticals explains that:
TGCT is caused by a genetic translocation in the colony-stimulating factor 1 (CSF1) gene resulting in overproduction of CSF1, triggering migration of inflammatory cells including CSF1R-expressing tumor-associated macrophages (TAMs) to tumor sites.
Vimseltinib may be an option for TGCT because it is:
an investigational, orally administered, potent and highly selective switch-control kinase inhibitor of CSF1R discovered using our proprietary drug discovery platform and designed to selectively bind to the CSF1R switch pocket.
123 participants with TGCT enrolled in the first portion of the Phase 3 MOTION trial. Participants received either a placebo or 30mg vimseltinib 2x weekly for a 24-week period. The second portion of the study, in which both groups are treated with vimseltinib, is still ongoing.
Results from the first portion of the study (which has a data cutoff of August 22, 2023) show that:
- The objective response rate was 40% in those taking vimseltinib vs. 0% for those receiving the placebo.
- Vimseltinib showed statistically significant and clinically meaningful benefits when compared to the placebo.
- The trial met secondary endpoints including physical function, pain, quality-of-life improvements, and tumor volume score.
- There were no signs of hepatotoxicity related to treatment.
- Vimseltinib was safe and relatively well-tolerated. However, some side effects did occur. These included facial and/or periorbital edema, fatigue, rashes, muscle pain, abnormal weakness, headaches, itchiness, diarrhea, high blood pressure, and nausea. About 6% of the participants receiving vimseltinib discontinued treatment due to these reactions.
Moving forward, Deciphera Pharmaceuticals plans to submit a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in the European Union during the course of 2024.
About Tenosynovial Giant Cell Tumors (TGCT)
Tenosynovial giant cell tumors are rare, locally aggressive, and typically benign (non-cancerous) tumors that affect the bursae, synovium, and tendon sheath.
TGCT may be considered localized or diffuse. These might differ in clinical presentation and age of onset. Learn more through TGCT Support.
Symptoms may vary based on where the tumors are located. Potential symptoms may include:
- A locking sensation in the joints
- Pain, swelling, and warmth in the affected area
- Limited range of motion
- Joint instability
- Stiffness
- “Clicking” or “popping” joints
Without treatment, TGCT can cause damage and degeneration, which may significantly impair quality-of-life.
