In final data from the Phase III ENLIVEN study, Turalio (pexidartinib) demonstrated durable tumor responses and a consistent safety profile in patients with symptomatic TGCT not amenable to surgery.
Daiichi Sankyo has shared final long-term data from the Phase III ENLIVEN clinical trial (NCT02371369). In the study, treatment with Turalio (pexidartinib) showed a sustained clinical benefit in patients with symptomatic tenosynovial giant cell tumor (TGCT) not amenable to improvement with surgery.1
In a press release, Andrew Wagner, MD, PhD, medical oncologist, Dana-Farber Cancer Institute and Harvard Medical School, said: “Prior to the approval of Turalio, the first FDA-approved systemic therapy for TGCT, there were limited treatment options beyond additional surgery. The final results of the ENLIVEN trial show the potential for long-term tumor responses with Turalio treatment with a safety profile consistent with earlier findings.”
Final long-term results from the second part of the ENLIVEN study showed:
Additionally, the safety profile of Turalio was consistent with previous findings from the first part of ENLIVEN, with no new safety signals identified.
In the press release, Patricia Judson, MD, vice president, US medical affairs, Daiichi Sankyo, added: “The final results of ENLIVEN contribute to the body of evidence supporting the long-term benefit of Turalio. Daiichi Sankyo is proud to have led the discovery and development of the first FDA-approved oral medicine for this rare disease. Since its approval nearly six years ago, more than 750 patients in the US have been treated with Turalio and we remain committed to working closely with healthcare professionals to help identify appropriate patients who may benefit from this treatment.”
Turalio was approved by the FDA in August 2019 for adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery. The approval was based on durable ORR observed earlier in ENLIVEN.2
At the time of the approval, positive results from the trial showed:
ENLIVEN is a global, double-blind, randomized, placebo-controlled study. In the first part, 120 patients were randomized to receive either Turalio at 1,000 mg daily for two weeks followed by 800 mg daily for 22 weeks or matching placebo.
Following completion of the first part of the trial, patients were offered to enter a long-term, open-label portion. In this second part of the study, 91 patients either crossed over from placebo to receive Turalio at 800 mg twice daily (without loading dose) or continued the dose of Turalio received at the end of the first part of the trial until tumor progression, toxicity, or study completion.
1. TURALIO® Final Long-Term Data Showed Sustained Clinical Benefit in Patients with Tenosynovial Giant Cell Tumor from Open-Label Extension of ENLIVEN Phase 3 Trial. News release. Daiichi Sankyo. July 9, 2025. Accessed July 9, 2025. https://www.businesswire.com/news/home/20250709712562/en/TURALIO-Final-Long-Term-Data-Showed-Sustained-Clinical-Benefit-in-Patients-with-Tenosynovial-Giant-Cell-Tumor-from-Open-Label-Extension-of-ENLIVEN-Phase-3-Trial
2. FDA approves pexidartinib for tenosynovial giant cell tumor. FDA. Accessed July 9, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pexidartinib-tenosynovial-giant-cell-tumor
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