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PEXIDARTINIB
At a glance
| Generic name | PEXIDARTINIB |
|---|---|
| Modality | Small molecule |
| Phase | FDA-approved |
| First approval | 2019 |
Approved indications
Boxed warnings
- WARNING: HEPATOTOXICITY TURALIO can cause serious and potentially fatal liver injury, including vanishing bile duct syndrome [see Warnings and Precautions (5.1) ] . Monitor liver tests prior to initiation of TURALIO and at specified intervals during treatment. Withhold and dose reduce or permanently discontinue TURALIO based on severity of hepatotoxicity. Monitoring and prompt cessation of TURALIO may not eliminate the risk of serious and potentially fatal liver injury [see Dosage and Administration (2.2) , Warnings and Precautions (5.1) ] . TURALIO is available only through a restricted program called the TURALIO Risk Evaluation and Mitigation Strategy (REMS) Program [see Warnings and Precautions (5.2) ] . WARNING: HEPATOTOXICITY See full prescribing information for complete boxed warning. TURALIO can cause serious and potentially fatal liver injury, including vanishing bile duct syndrome. ( 5.1 ) Monitor liver tests prior to initiation of TURALIO and at specified intervals during treatment. Withhold and dose reduce or permanently discontinue TURALIO based on severity of hepatotoxicity. Monitoring and prompt cessation of TURALIO may not eliminate the risk of serious and potentially fatal liver injury. ( 2.2 , 5.1 ) TURALIO is available only through a restricted program called the TURALIO Risk Evaluation and Mitigation Strategy (REMS) Program. ( 5.2 )
Common side effects
- Hair color changes
- Fatigue
- Increased lactate dehydrogenase
- Increased aspartate aminotransferase
- Increased alanine aminotransferase
- Decreased neutrophils
- Increased cholesterol
- Increased alkaline phosphatase
- Decreased lymphocytes
- Eye edema
- Decreased hemoglobin
- Rash
Key clinical trials
- Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ... (PHASE1)
- I-SPY TRIAL: Neoadjuvant and Personalized Adaptive Novel Agents to Treat Breast Cancer (PHASE2)
- A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan (PHASE2)
- A Study of PLX3397 in Patients With Unresectable or Metastatic KIT-mutated Melanoma (NA)
- A Long-term Study Evaluating Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment
- A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT (PHASE3)
- PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors (PHASE1, PHASE2)
- CGT9486 (Formerly Known as PLX9486) as a Single Agent and in Combination With PLX3397 (Pexidartinib) or Sunitinib in Participants With Advanced Solid Tumors (PHASE1, PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- PEXIDARTINIB CI brief — competitive landscape report
- PEXIDARTINIB updates RSS · CI watch RSS