Turalio (pexidartinib)
An approved treatment for Tenosynovial Giant Cell Tumor.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Pexidartinib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Pexidartinib
- Brand name
- Turalio
- Development code
- PLX3397
- Drug class
- CSF1R kinase inhibitor
- Manufacturer
- Daiichi Sankyo
- How it's taken
- Oral capsule, twice a day with a low-fat meal
A capsule from Daiichi Sankyo taken twice a day for adults with tenosynovial giant cell tumor (TGCT) that causes severe symptoms and cannot be improved with surgery. The FDA approved it on August 2, 2019 as the first medicine for TGCT. It carries a boxed warning for liver injury and is available only through a REMS program.
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Where Pexidartinib fits
Surgery is the usual first treatment for TGCT, and Turalio is for people whose tumor cannot be helped by surgery. Romvimza (vimseltinib), approved in 2025, blocks the same receptor and is taken twice a week without a boxed warning.
How Pexidartinib works
Tenosynovial giant cell tumor grows in the lining of a joint or tendon. A small group of tumor cells makes too much of a signal called CSF1, which draws in the inflammatory cells that make up most of the tumor. Pexidartinib blocks CSF1R, the receptor that signal acts on, so the tumor can shrink.
Side effects and safety
- Liver. Of the first 609 people treated under the REMS program, 32 (5.3%) had a serious liver event, all within 71 days of the first dose.
- REMS program. Prescribers, patients and pharmacies must all enroll in the Turalio REMS; details at www.TURALIOREMS.com or 1-833-887-2546.
- Pregnancy. Can harm an unborn baby. Women are advised to use non-hormonal birth control during treatment and for 1 month after, because Turalio can stop hormonal birth control from working.
- High-fat meals. Taking it with a high-fat meal raises drug levels and may increase side effects, including liver injury.
- Liver tests before starting, weekly for the first 8 weeks, every 2 weeks for the next month, then every 3 months
- A pregnancy test before starting for anyone who could become pregnant
Turalio carries a boxed warning for serious and potentially fatal liver injury, including vanishing bile duct syndrome, a condition where the small bile ducts in the liver are lost. Of the first 609 people treated under the REMS program, 32 (5.3%) had a serious liver event, and all 32 began within 71 days of the first dose. In the ENLIVEN trial, the most common side effects on Turalio compared with placebo were hair color changes (67% against 3.4%), fatigue (64% against 41%), swelling around the eyes (30% against 5%) and rash (28% against 7%).
Changes in taste were also more common (26% against 1.7%). Serious side effects occurred in 13% of people on Turalio, and 13% stopped because of side effects. It can harm an unborn baby, and the label advises non-hormonal birth control because Turalio can stop hormonal methods from working.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Pexidartinib
Take 250mg by mouth twice a day with a low-fat meal of about 11-14g of fat. A high-fat meal raises drug levels and may increase side effects, including liver injury. Capsules are swallowed whole and never opened, broken or chewed. After a missed or vomited dose, the next dose is taken at its usual time. If side effects need it, the dose can be lowered to 125mg in the morning and 250mg in the evening, then to 125mg twice a day.
Availability and cost
Only available as the brand-name product.
Help paying for Turalio
Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE. Pick your insurance below to see which offers fit you.
Good to know: Daiichi Sankyo AccessCentral4U covers Turalio, with help on insurance coverage and access, financial assistance, and medicine at no cost for eligible uninsured or underinsured patients. Turalio is dispensed only through the Turalio REMS program.
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Access and eligibility
Approved for adults with symptomatic TGCT linked to severe morbidity or functional limitation that surgery cannot improve. Prescribers, patients and pharmacies must all enroll in the Turalio REMS, and only certified pharmacies can dispense it.
Source: Turalio REMS
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
ENLIVEN (NCT02371369) was a Phase 3, double-blind trial that randomized 120 adults with TGCT not suited to surgery, 61 to Turalio and 59 to placebo. Most had the diffuse form (88%), and the knee was the most common site (61%). By week 25, tumors shrank by the standard RECIST measure in 38% of people on Turalio (15% completely) against 0% on placebo.
Joint range of motion also improved more on Turalio. The trial used a higher dose on an empty stomach, and the label says the approved 250mg dose with a low-fat meal gives similar drug levels.
Development history
Plexxikon, later part of Daiichi Sankyo, developed pexidartinib under the code PLX3397. The FDA approved Turalio on August 2, 2019, making it the first approved medicine for TGCT, with the REMS program required from the start because of liver injury seen in trials. Daiichi Sankyo also runs a study following liver safety in people taking Turalio (NCT04635111).
Explore Tenosynovial Giant Cell Tumor trials
Other Tenosynovial Giant Cell Tumor treatments
Common questions about Pexidartinib
▸What is Pexidartinib (Turalio)?
A capsule from Daiichi Sankyo taken twice a day for adults with tenosynovial giant cell tumor (TGCT) that causes severe symptoms and cannot be improved with surgery. The FDA approved it on August 2, 2019 as the first medicine for TGCT. It carries a boxed warning for liver injury and is available only through a REMS program.
▸How does Pexidartinib work?
Tenosynovial giant cell tumor grows in the lining of a joint or tendon. A small group of tumor cells makes too much of a signal called CSF1, which draws in the inflammatory cells that make up most of the tumor. Pexidartinib blocks CSF1R, the receptor that signal acts on, so the tumor can shrink.
▸What are the side effects of Pexidartinib?
Turalio carries a boxed warning for serious and potentially fatal liver injury, including vanishing bile duct syndrome, a condition where the small bile ducts in the liver are lost. Of the first 609 people treated under the REMS program, 32 (5.3%) had a serious liver event, and all 32 began within 71 days of the first dose. In the ENLIVEN trial, the most common side effects on Turalio compared with placebo were hair color changes (67% against 3.4%), fatigue (64% against 41%), swelling around the eyes (30% against 5%) and rash (28% against 7%). Changes in taste were also more common (26% against 1.7%). Serious side effects occurred in 13% of people on Turalio, and 13% stopped because of side effects. It can harm an unborn baby, and the label advises non-hormonal birth control because Turalio can stop hormonal methods from working.
▸How is Pexidartinib taken?
Take 250mg by mouth twice a day with a low-fat meal of about 11-14g of fat. A high-fat meal raises drug levels and may increase side effects, including liver injury. Capsules are swallowed whole and never opened, broken or chewed. After a missed or vomited dose, the next dose is taken at its usual time. If side effects need it, the dose can be lowered to 125mg in the morning and 250mg in the evening, then to 125mg twice a day.
▸Is Pexidartinib FDA approved?
Yes, Pexidartinib (Turalio) is FDA approved (2019) for the treatment of Tenosynovial Giant Cell Tumor.
▸What is Turalio used for?
Turalio (pexidartinib) is FDA-approved for adults with tenosynovial giant cell tumor (TGCT) that causes severe symptoms or limits function and cannot be improved with surgery.
▸Why does Turalio have a REMS program?
Turalio can cause serious and potentially fatal liver injury, so the FDA requires prescribers, patients and pharmacies to enroll in the Turalio REMS. Patients sign an enrollment form and get liver tests on a set schedule.
▸How well did Turalio work in the ENLIVEN trial?
By week 25, tumors shrank by the standard RECIST measure in 38% of people on Turalio and in none on placebo, and 15% saw the tumor disappear on scans, according to the label.
▸How is Turalio different from Romvimza?
Both block CSF1R and are approved for TGCT. Turalio is taken twice a day with a low-fat meal and carries a boxed warning for liver injury, while Romvimza (vimseltinib) is taken twice a week and has no boxed warning, according to their labels.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2025-01. TURALIO (pexidartinib) capsules: Prescribing Information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7b0ec4bf-b2ac-49ee-96af-7740ad3f2e16
- U.S. Food and Drug Administration · 2019-08-02. FDA approves first therapy for rare joint tumor. https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-joint-tumor
- Daiichi Sankyo, Inc.. Patient Assistance Program - Access Central. https://daiichisankyo.us/patient-assistance-programs
- Daiichi Sankyo. TURALIO Risk Evaluation and Mitigation Strategy (REMS) Program. https://www.turaliorems.com/
- ClinicalTrials.gov. ENLIVEN: Phase 3 Study of Pexidartinib in Tenosynovial Giant Cell Tumor. https://clinicaltrials.gov/study/NCT02371369