Turalio (pexidartinib) capsules have been approved for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) that is associated with severe morbidity or functional limitations not responsive to improvement with surgery, the U.S. Food and Drug Administration announced.
Turalio is the first therapy to be approved for the rare joint tumor and is available only through the Turalio Risk Evaluation and Mitigation Strategy (REMS) Program. The FDA granted the approval of Turalio to Daiichi Sankyo.
“TGCT can cause debilitating symptoms for patients such as pain, stiffness and limitation of movement,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a. “Surgery is the primary treatment option, but some patients are not eligible for surgery, and tumors can recur, even after the procedure.”
The approval was based on results of a study of 120 patients, 59 of whom received placebo. After 25 weeks of treatment, the overall response rate was 38% (15% complete responses and 23% partial responses) in those who received pexidartinib; no responses occurred in patients who received placebo. The response persisted in 22 of 23 responders who had been followed for a minimum of 6 months, and in 13 of 13 responders who had been followed for a minimum of 12 months.
Turalio comes with a Boxed Warning about the risk of serious and potentially fatal liver injury. Liver tests should be performed prior to beginning treatment and the results monitored at specified intervals during treatment. Patients who develop abnormal results may need to withhold therapy, reduce the dose, or discontinue therapy depending on the severity of the liver injury.
Common side effects for patients were increased levels of lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase, and cholesterol. Loss of hair color also occurred in some patients.
Additional side effects included neutropenia, increased alkaline phosphatase levels, decreased lymphocytes, eye edema, decreased hemoglobin levels, rash, dysgeusia, and decreased phosphate levels.
Females of reproductive age and males with a female partner of reproductive potential should use effective contraception during treatment with pexidartinib. Pexidartinib may cause harm to a developing fetus or newborn baby.
Pexidartinib must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.