The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for pexidartinib (Daiichi Sankyo) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT). 

Pexidartinib is an oral small molecule that is believed to work by inhibiting colony stimulating factor-1 receptor (CSF1R), the primary growth driver of abnormal cells in the synovium that cause TGCT. The NDA is supported by data from the Phase 3 ENLIVEN study (N=120) which randomized patients with symptomatic TGCT to receive pexidartinib or placebo to evaluate the safety and efficacy of the treatment. Results showed at Week 25, the overall response rate (primary endpoint) for the intention-to-treat population was 39.3% in the pexidartinib arm vs 0% for placebo (P< .0001).

With regard to safety, hepatic toxicities were more frequent with pexidartinib vs placebo; 8 patients in the pexidartinib arm discontinued due to hepatic effects. Other adverse events included hair color changes, vomiting, fatigue, dysgeusia, and periorbital edema. 

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“Current treatment options for TGCT are largely limited to surgery, but for some patients the disease is debilitating and not amenable to improvement with surgery,” said Dale Shuster, PhD, Executive Director, Global Oncology R&D, Daiichi Sankyo. “We are committed to working with the FDA to potentially bring pexidartinib to carefully-selected patients as soon as possible.”

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