FDA Approves Turalio to Treat Tenosynovial Giant Cell Tumors

The Food and Drug Administration (FDA) approved Turalio (pexidartinib) to treat tenosynovial giant cell tumors (TGCT) — a rare type of disease, according to an FDA press release.

Tenosynovial giant cell tumors are a group of rare, benign tumors that involve the synovium (the thin layer of tissue or membrane that covers the inner surface of the joint spaces and the bursae and tendon sheaths), bursae (small fluid-filled sacs that cushion bones, tendons, and muscles around the joints) and tendon sheath (fibrous tissue that connect muscle to bone).

The disease can be locally aggressive and debilitating, and is associated with severe morbidity or function limitations. Prior to today’s approval, surgery was the primary treatment for patients with TGCT, with no FDA-approved systemic therapies.

“TGCT can cause debilitating symptoms for patients such as pain, stiffness and limitation of movement. The tumor can significantly affect a patient’s quality of life and cause severe disability,” said Dr. Richard Pazdur, 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. “Surgery is the primary treatment option, but some patients are not eligible for surgery, and tumors can recur, even after the procedure. Today’s approval is the first FDA-approved therapy to treat this rare disease.”

Turalio — which was also mentioned in the American Society of Clinical Oncology’s January 2019 announcement of selecting progress in treating rare cancers as its Advance of the Year – was granted priority review in February based on results from the phase 2 ENLIVEN study showing improved responses to the agent. In May, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 12 to 3 in favor of Turalio for an indication as a treatment of adult patients with the disease.

Check back next week for more information about the approval.