Today, Daiichi Sankyo Company announced that its new drug, pexidartinib, significantly reduced the tumor size of patients in phase 3 clinical trials and reached the primary clinical endpoint. The results of this clinical trial support its potential to treat tenosynovioblastoma (TGCT).
Usually Tendon sheath giant cell tumor is a benign tumor that appears in joints and soft tissues. The disease can be divided into two categories. Local tumor is often inert, while the diffuse tumor is more invasive. Because of the delicate location of the disease, the risk of surgery is higher, and the injury for the patient is often greater than the advantage. Therefore, if there is a treatment that does not throuth the operation, it will bring good improvement to the patient's life.
Pexidartinib is the new drug that promises to benefit patients. It is a CSF1R inhibitor . Previous studies have found that ganglion tomoblastoma is driven by over expression of CSF1. Therefore, pexidartinib is expected to control the occurrence of disease from its roots.
▲ Molecular structure of Pexidartinib (image source: Selleck Chemicals)
In the phase 3 clinical trial called ENLIVEN, the researchers assessed the therapeutic potential of pexidartinib. The trial was divided into two groups, one receiving treatment with pexidartinib and the other receiving a placebo. After 24 weeks, researchers will use techniques such as MRI to assess the patient's recovery. Patients who have completed this stage are eligible to enter the next long-term study and receive the pexidartinib treatment with open labelling.
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