Skip to main content
CTOS 2025 Main banner
Final Program


Schedule at a Glance
Icon Legend
This session is not in your schedule.
This session is in your schedule. Click again to remove it.

Soft Tissue Tumors

(P 336) PEXIDARTINIB RISK EVALUATION AND MITIGATION STRATEGY PROGRAM: 5-YEAR RETROSPECTIVE HEPATIC SAFETY ASSESSMENT

    Author(s)

  • Abdul Waheed Rajper, MD

    Director Clinical Safety
    Clinical Safety & Pharmacovigilance, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA
    Basking Ridge, New Jersey, United States

    • Co-Author(s)

  • Charles Dharmani, DrPH, PharmD, MPH, MS

    Senior Director, Epidemiology, Clinical Safety & Pharmacovigilance
    Daiichi Sankyo, Inc., Basking Ridge, NJ, USA
    Basking Ridge, New Jersey, United States

  • LC

    Lalitha Chilakapati, PharmD

    Clinical Safety & Pharmacovigilance, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA
    Basking Ridge, New Jersey, United States

  • OF

    Oluwatosin Fofah, MD

    Epidemiology, Clinical Safety and Pharmacovigilance, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA
    Basking Ridge, New Jersey, United States

  • SC

    Sujani Chitneedi, Mpharm

    Clinical Safety & Pharmacovigilance, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA
    Basking Ridge, New Jersey, United States

  • KT

    Kristen Tecson, PhD

    Evidence Generation, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA
    Basking Ridge, New Jersey, United States

  • MW

    Margaret J. Wooddell, PhD, MBA

    Global Oncology Medical Affairs, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA
    Basking Ridge, New Jersey, United States

Objective: First-in-class TKI pexidartinib (pexi; TURALIO®) is conditionally approved by the US FDA for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) based on the development of the TURALIO® REMS (tREMS) Program. We report hepatic safety data of pexi in adult patients (pts) with TGCT from the retrospective 5-year evaluation of the tREMS Program.

Methods: Cumulative hepatic data were collected from Aug 2019 to Jun 2024 from healthcare practitioners, pts, pharmacies, and distributors who participated in the tREMS Program. Liver AE reporting forms captured data for pts who experienced a liver AE or serious or potentially fatal liver injury. For liver injury events of concern (LIEC), data were manually extracted in May 2025 from the global safety database and pts narratives. All data were summarized using descriptive statistics.

Results: 784 pts were enrolled in the tREMS Program; 735 received ≥1 dispense of pexi. There were 45 pts (6.1%) with a LIEC (Table). The median (Q1, Q3) time to event onset was 27 d (21, 48). Most pts (73.3%) were on the same daily dose at the time of event compared with their initial dose; 46.7% and 24.4% of pts were taking a daily dose of 800 mg or 500 mg, respectively, at the time of event onset, reflective of the dose-equivalent formulations: 200-mg tablet (empty stomach) and 125-mg tablet (low-fat meal). The median (Q1, Q3) time to event onset was similar for patients taking a daily dose of 800 mg (22.5 d [18.5, 27.5]) at event onset and for pts taking 500 mg (29 d [19, 44]). As of last follow-up (Feb 2025), 71.1% of pts recovered, 13.3% were recovering, 13.3% had not yet recovered, and 2.2% had an unknown status.

Conclusion: The results from this retrospective 5-year hepatic safety assessment of the tREMS Program show that careful monitoring of liver enzyme levels, along with early intervention, can mitigate the risk of potential hepatotoxicity associated with pexi treatment in adult pts with TGCT. The frequency, pattern, and timing of serious hepatic AEs was consistent with previous reports, and no new safety signals were observed.

Upload Image