Drug updated on 4/18/2024
| Dosage Form | Capsule (oral; 125 mg) |
| Drug Class | Kinase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery.
Summary
- Turalio (Pexidartinib) is indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. It demonstrates a modest improvement in pain relief over placebo, significant enhancements in physical function and stiffness, and exhibits a robust tumor response.
- The insights were derived from four Randomized Controlled Trials that provide insights into Turalio's safety, effectiveness, and comparative positioning relative to placebo or the absence of an alternative systemic therapy for TGCT.
- One study demonstrates a critical correlation between tumor size reduction and improvement in pain scores, suggesting Turalio's benefits may be closely tied to its effectiveness in reducing tumor burden.
- Sustained beneficial effects on physical function and reduction in stiffness underscore Turalio's potential to enhance the quality of life for TGCT patients who cannot undergo surgery.
- A population pharmacokinetic analysis reveals modest differences based on demographic factors such as race, sex, body weight, etc., implying stability across various patient subgroups.
- While the drug has shown a significantly higher response rate compared to placebo, hepatotoxicity has been identified as a major concern regarding the drug’s safety profile, necessitating vigilant monitoring.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Turalio (pexidartinib) Prescribing Information. | 2023 | Daiichi Sankyo Inc., Basking Ridge, NJ |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Pexidartinib provides modest pain relief in patients with tenosynovial giant cell tumor: results from ENLIVEN. | 120Subjects F: 59% M: 41% | 2023 | Clinical Orthopaedics and Related Research |
| Pexidartinib improves physical functioning and stiffness in patients with tenosynovial giant cell tumor: results from the ENLIVEN randomized clinical trial. | 120Subjects F: 59% M: 41% | 2021 | Acta Orthopaedica |
| Population pharmacokinetic analysis of pexidartinib in healthy subjects and patients with tenosynovial giant cell tumor or other solid tumors. | 375Subjects F: 38% M: 62% | 2021 | Journal of Clinical Pharmacology |
| Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. | 120Subjects F: 51% M: 49% | 2019 | The Lancet |
Document Title
Sex Distribution:
F:59%
M:41%
120Subjects
Year:
2023
Source:Clinical Orthopaedics and Related Research
Sex Distribution:
F:59%
M:41%
120Subjects
Year:
2021
Source:Acta Orthopaedica
Sex Distribution:
F:38%
M:62%
375Subjects
Year:
2021
Source:Journal of Clinical Pharmacology
Document Title
Sex Distribution:
F:51%
M:49%
120Subjects
Year:
2019
Source:The Lancet