Approval was granted today by the United States Food and Drug Administration to Turalio (pexidartinib) capsules for treating the adult patients with symptomatic tenosynovial giant cell tumor associated with severe functional limitations or morbidity and not receptive to improvement with surgery.
The Acting Director of the Office of Hematology & Oncology Products in the FDA’s Center for Drug Evaluation and Research, and the Managing Director of the Food & Drug Administration’s Oncology Center of Excellence, Richard Pazdur stated that tenosynovial giant cell tumor can cause debilitating symptoms for patients such as stiffness, pain, & limitation of movement. The tumor can affect a patient’s quality of life significantly & cause severe disability. Surgery is the major treatment option, however, some of the patients aren’t eligible for surgery, & tumors can recur, even after the procedure. The approval which received today is the first FDA-approved therapy for the treatment of this rare disease.
Tenosynovial giant cell tumor is an unusual tumor which affects the synovium, which is the thin layer of tissue that covers the surfaces of the joint spaces, & tendon sheaths, which are fibrous tissue that connects muscle to bone. The tumor is malignant is very rare cases but causes the synovium & tendon sheaths to overgrow and thicken, causing damage to surrounding tissue.
The approval of this therapy was based on the outcomes of a multi-center international clinical trial of 120 patients, 59 amongst whom received a placebo. The key efficacy endpoint was the overall response rate analyzed after twenty-five weeks of treatment. The clinical trial gave a demonstration of a statistically significant improvement in overall response rate in patients who received Turalio, with an overall response rate of 38 percent as compared with no responses in patients who received a placebo.
Medics are advised by the Food & Drug Administration to tell females of reproductive age & males with a female partner of reproductive potential to use effective contraception during treatment with Turalio. Women who are breastfeeding or pregnant should not take Turalio because it might cause harm to a developing fetus or newborn baby.