FDA approves Turalio to treat symptomatic tenosynovial giant cell tumor

Daiichi Sankyo Company, Limited announced that the FDA approved Turalto (pexidartinib) as the first and only treatment for adult patients with symptomatic TGCT (symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery.

TGCT is a rare, non-malignant tumor that affects small and large joints. The disease can cause debilitating symptoms and can be locally aggressive.The FDA approval of Turalio is based on the results of the pivotal phase III ENLIVEN study, the first placebo-controlled study of a systemic therapy in patients with TGCT. Study results showed the primary endpoint of tumor response rate by Response Evaluation Criteria v1.1 in Solid Tumors (RECIST) was 38 percent (95% CI: 27%, 50%) in Turalio treated patients and zero percent (95% CI: 0%, 6%) for placebo-treated patients at Week 25 (Turalio N=61, placebo N=59; p<0.0001). In addition, overall response rate by tumor volume score (TVS) was 56 percent (95% CI: 43%, 67%) in patients randomized to the Turalio arm and zero percent in patients randomized to the placebo arm at Week 25 (Turalio N=61, placebo N=59; p<0.0001). Furthermore, the analysis of mean change from baseline in range of motion at Week 25 (Turalio N=45, placebo N=43) demonstrated a statistically significant improvement in patients treated with Turalio, compared to placebo.