Pexidartinib
Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.
| ID | DRUG-PEXIDARTINIB |
|---|---|
| Type | Drug |
| Aliases | TuralioПексидартиніб |
| Status | reviewed 2026-05-04 | pending_clinical_signoff |
| Diseases | DIS-TGCT |
| Sources | SRC-ESMO-SARCOMA-2024 SRC-ILLUMINATE-TAP-2019 |
Drug Facts
| Class | CSF1R inhibitor (oral, selective) |
|---|---|
| Mechanism | Selective oral inhibitor of CSF1R (colony-stimulating factor 1 receptor), KIT, and FLT3. In TGCT (formerly PVNS), tumor cells overexpress CSF1 (via COL6A3-CSF1 translocation), driving recruitment of CSF1R-expressing macrophages and osteoclast-like giant cells that constitute the bulk of the tumor mass. Pexidartinib depletes these CSF1R+ cells, reducing tumor burden. ILLUMINATE trial: ORR 38% (vs 0% placebo), with 15% complete response. FDA-approved August 2019 — first systemic therapy for TGCT. |
| Typical dosing | 400 mg PO twice daily with a high-fat meal for the first 2 weeks (loading phase), then 800 mg/day total — consult current FDA prescribing information. Available through REMS program only. |
| Ukraine registered | False |
| NSZU reimbursed | False |
| Ukraine last verified | 2026-05-04 |
Warnings
- Serious hepatotoxicity including fatal cases — available only through REMS program (TURALIO REMS); monitor LFTs weekly for 2 months, then monthly
Notes
ILLUMINATE trial (Tap 2019 NEJM): pexidartinib vs placebo in symptomatic TGCT (n=120, crossover design). ORR 38% (pexidartinib) vs 0% (placebo); CR 15%. Response was durable (median DOR not reached). Hepatotoxicity was the key safety signal — 5% G3/4 ALT/AST elevation; serious hepatotoxicity including cases requiring transplant. REMS (Risk Evaluation and Mitigation Strategy) program required in the US — restricted distribution, mandatory LFT monitoring, patient enrollment. EMA refused marketing authorization due to hepatotoxicity concerns. Reserved for adults with symptomatic TGCT not amenable to improvement with surgery alone.
Used By
Regimens
REG-PEXIDARTINIB-TGCT- Pexidartinib monotherapy (symptomatic TGCT — diffuse-type)