In tenosynovial giant cell tumor (TGCT), a small number of cells that line the joints can have a defect that causes them to make too much of a protein called colony-stimulating factor 1 (CSF-1). CSF-1 acts like a magnet and attracts inflammatory cells into the joint space to form a tumor. TURALIO™ (pexidartinib) blocks the signals of CSF-1.
TURALIO was studied in a clinical trial of 120 patients who had advanced TGCT, for whom surgery was not recommended. For 24 weeks, half of the patients in the trial were given TURALIO and half were given a placebo.* After 24 weeks, patients in either group could continue in the study and receive TURALIO.
*A placebo (or “sugar pill”) contains no active ingredient and provides no medical effect.
Of patients in the trial:
• 88% (105 of 120 patients) had diffuse TGCT.
• 47% (56 of 120 patients) had never had and were not eligible for surgery for TGCT.
TURALIO: Clinical Trial Results
None of the patients taking placebo experienced a reduction in tumor length or volume.
A reduction in tumor size meant that tumors decreased by at least 30% in length. A reduction in tumor volume meant that tumors decreased by at least 50% in volume.
TURALIO improved range of motion
After a clinical assessment at 25 weeks, results showed that patients taking TURALIO had significantly improved range of motion compared to patients taking placebo. Results were not available for 32 patients.
Serious liver problems: TURALIO can cause serious injury to the liver, which can be severe and lead to death.
Before starting treatment, your doctor will do blood tests to check for liver problems. These tests will be repeated while you are taking TURALIO. If you develop liver problems during treatment with TURALIO, your healthcare provider may do blood tests more often to monitor you. It is important to stay under the care of your healthcare provider during treatment with TURALIO.
Stop taking TURALIO and call your healthcare provider right away if you develop:
- yellowing of your skin and whites of your eyes
- dark urine
Tell your healthcare provider right away if you have any of these symptoms of liver problems while taking TURALIO:
- lack or loss of appetite
- right upper stomach-area (abdomen) pain or tenderness
- feeling overly tired
- changes in blood liver tests
- hair color changes
- increased cholesterol level in your blood
- decreased white blood cells and red blood cells
- swelling in or around your eyes
- loss of taste or changes in the way things taste
TURALIO may affect fertility in females and males, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility.
These are not all of the possible side effects of TURALIO. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or fda.gov/medwatch. You may also report side effects to Daiichi Sankyo, Inc. at 1-877-4DS-PROD (1-877-437-7763).
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