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the U.S. Food and Drug Administration (FDA) approved Dayichi Sankyo's Pexidartinib capsule for the treatment of adult patients with tendon cytomoma (TGCT) symptoms, who are associated with severe morbidity or functional limitations and have not improved through surgery.
The approval was based on the results of a multi-center international clinical trial of 120 patients and found a significant improvement in overapp response (ORR) statistics for patients receiving Turalio, with an ORR of 38,
percent
. Patients receiving a placebo did not respond. The full mitigation rate is 15%, and the partial mitigation rate is 23
%
.
"TGCT can cause 'debilitating symptoms, such as pain, stiffness, and limited movement' in the patient's body." Tumors can significantly affect a patient's quality of life and lead to severe disability," said Richard Pazdur, director of the FDA Center of Excellence in Oncology at the FDA Drug Evaluation and Research Center and acting director of hematology and oncology products.
He continued, "Surgery is the primary treatment option, but some patients are not eligible for surgery, and even after surgery the tumor will return." Today's approval is the first FDA-approved treatment for this rare disease. GCT is a rare tumor that affects the slip membrane (a thin layer of tissue that covers the surface of the joint gap) and a tendon that covers the membrane layer of the tendon, the fibrous tissue that connects the muscles to the bones. Tumors are rarely malignant but cause the membranes and tendons to thicken and overgrow, causing damage to surrounding tissue. (cyy123.com)