Daiichi Sankyo's Oral Anticancer Drug Pexidartinib Capsules Recommended for Priority Review in China for TGCT

Oct 24, 2024 17:06 CST Updated 17:06

Daiichi-Sankyo

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Today (October 24), the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has just announced,Daiichi Sankyo's PLX3397 (Pexidartinib Hydrochloride) Capsules Proposed for Priority Review in ChinaAdult patients with symptomatic tenosynovial giant cell tumor (TGCT) accompanied by severe lesions or functional limitations that cannot be improved through surgery.。Public information shows,Pexidartinib (pexidartinib）It is a CSF1R small molecule inhibitor, which was previously approved for marketing by the US FDA in August 2019 to treat adult patients with symptomatic tenosynovial giant cell tumor (TGCT). According to the FDA's press release at that time, this is the first treatment method for TGCT that has been approved.

Screenshot source:CDE Official Website

Tenosynovial Giant Cell Tumor (TGCT), also known as Pigmented Villonodular Synovitis, is a rare non-malignant tumor with local invasiveness.It can affect synovial-covered joints, bursae, and tendon sheaths, leading to swelling, pain, stiffness in the joints or limbs, as well as a decreased range of motion.Currently, surgery is the standard treatment for tenosynovial giant cell tumors (TGCT). However, for patients with recurrent, refractory, or diffuse TGCT,Surgery is not only difficult for removing the tumor, but may also lead to serious joint damage and loss of function.For some patients, they may consider amputation to relieve pain.。

Pexidartinib is an innovative oral small-molecule CSF1R inhibitor,It also inhibits the activity of c-kit and FLT3-ITD.。The CSF1R-mediated signaling pathway is a key driver of abnormal cell proliferation in the synovium.In the United States, pexidartinib has been granted Breakthrough Therapy designation, Orphan Drug status, and Priority Review by the FDA.

In August 2019, the FDA approved pexidartinib for the treatment of adult patients with symptomatic TGCT. This approval was based on the results of a multicenter international phase 3 clinical trial named ENLIVEN. A total of 120 patients received either pexidartinib (n=61) or placebo (n=59). The trial results showed,39% of patients treated with pexidartinib achieved remission after 25 weeks of treatment, compared to 0% in the placebo group.。The partial response rate was 23% and the complete response rate was 15%.Among the 23 patients who achieved remission, 22 maintained their remission at a follow-up of at least 6 months. Among the 13 patients with a follow-up period of at least 12 months, all patients had a remission duration exceeding 12 months.

According to the official website of the Chinese Drug Clinical Trial Registration and Information Disclosure Platform, Daiichi Sankyo has launched an international multicenter (including China) Phase 3 clinical study in China. This study aims to evaluate pexidartinib forAdult patients with symptomatic TGCT who have severe lesions or serious functional limitations that cannot be improved by surgeryThe efficacy and safety. This is also the indication proposed for priority review this time.

Looking forward to this innovationCSF1R small molecule inhibitors arrive to patients as soon as possible, bringing hope sooner.New Treatment Options for Patients with Giant Cell Tumors of the Tendon Sheath.

References:

[1] Center for Drug Evaluation, National Medical Products Administration of ChinaWebsite. Retrieved Oct 24, 2024, from https://www.cde.org.cn/main/xxgk/listpage/da6efd086c099b7fc949121166f0130c

[2]FDA approves first therapy for rare joint tumor. Retrieved August 2, 2019, from https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-joint-tumor

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