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

Published: 2025-07-09 09:05:31 ET 4568.T

Published on 07/09/2025 at 09:05

Final long-term efficacy and safety results from the open-label extension of the ENLIVEN phase 3 trial showed a sustained clinical benefit from long-term treatment with TURALIO®? (pexidartinib) in patients with symptomatic tenosynovial giant cell tumor (TGCT) not amenable to improvement with surgery. These results, consistent with the primary analysis of the trial, were recently published in The Oncologist.

TURALIO is the first oral systemic therapy approved in the U.S. for adult patients with TGCT associated with severe morbidity or functional limitations and not amenable to improvement with operations. TGCT is a rare and typically non-malignant tumor that affects small and large joints. Efficacy outcomes were measured by overall response rate (ORR) by RECIST version 1.1, ORR by tumor volume score (TVS) and mean change in baseline in range of motion of the affected joint at Week 25.

Study results from the first part of ENLIVEN showed an ORR of 38% (95% confidence interval [CI]: 27-50) in patients treated with TURALIO compared to an ORR of 0% (95% CI: 0-6) in patients treated with placebo as assessed by RECIST. An ORR by TVS of 56% (95% CI: 43-67) in patients treated with TurALIO and 0% in patients treated with placebo also was shown. A total of 91 patients received TURALIO during the second part of the study as of data cut-off of April 30, 2021.

Serious TEAEs were reported in 23.1% of patients who received TURALIO in the ENLIVEN study. Due to the risk of hepatotoxicity, TURALIO is only available through a restricted program called the TURALIO Risk Evaluation and Mitigation Strategy (REMS) Program. TURALIO can cause serious and potentially fatal liver injury, including devastating bile duct syndrome.

After completing the first part of the trial, patients randomized to either TURALIO or placebo were eligible to enter the second part of ENLIVEN, a long-term, open-label portion of the trial where 91 patients either crossed over from placebo to receive TURALIO 800 mg twice a day (without loading dose) or continued the dose of TURALIO received at the end of part 1 until tumor progression, toxicity, or study completion. Results from this portion of the trial were published in The Oncologist; TURALIO is available only through a restricted program called theTURALIO Risk Evaluation andMitigation Strategy (REMS) program. Of the first 609 patients who received TURALio under the REMS program, 32 (5.3%) developed a liver injury event of concern, defined as any serious liver-related outcome or any liver abnormality that triggers drug discontinuation per the US Prescribing Information.

Among 768 patients who received TURAL IO in clinical trials, there were two irreversible cases of cholestatic liver injury. To report a patient patient with TURALIO in the U.S. and the primary analysis of the trial was published in The Oncologist, Dana-Farber Cancer Institute and Harvard Medical School.