Making History: First Treatment for TGCT Gets FDA Approved

According to a story from fda.gov, the US Food and Drug Administration (FDA) has approved the drug pexidartinib (marketed as Turalio) as a treatment for tenosynovial giant cell tumor (TGCT) in patients experiencing symptoms that are inflicting mobility limitations or severe morbidity that have not responded successfully to surgical intervention. This is the first medication approved for this rare disease.

About Tenosynovial Giant Cell Tumor (TGCT)

Tenosynovial giant cell tumor (TGCT) is a disease which affects the joints, most typically the hip or knee. The illness is characterized by overgrowth of the joint lining and inflammation. Unfortunately the cause of this disease remains a mystery; prevailing theories include abnormal metabolism of fat, blood within the affected joint, or repeated inflammation. A risk factor is prior trauma affecting the joint where the disease appears. The symptoms of TGCT include sudden swelling and joint pain, loss of range of motion, progressive swelling and pain, bone erosion, and damage to surrounding tissues. The disease has the potential to spread from the affected joint and can lead to permanent disability and chronic pain. Treatment generally includes the surgical removal of the joint lining, radiation therapy, or replacement of the affected joint. TGCT can easily recur. To learn more about this disease, click here.

Breaking New Ground

While some patients respond well to surgery, recurrence of the condition is commonplace, and some patients are not eligible for surgery at all. Now, these patients who previously had no other recourse have an all new treatment option that can help improve their symptoms. Turalio was approved following the results of a clinical study that included a total of 120 TGCT patients. The overall response rate in this trial was 38 percent. Of this group, 15 percent experienced a complete response and 23 percent saw a partial response.

Monitoring of responses indicated that they were durable and long lasting; 22 of 23 patients that were monitored for six months maintained their response and 13 of 13 patients that were monitored for a year maintained their response. Turalio carries a risk of serious and possibly lethal liver injury and will include a boxed warning for this risk.

This drug was approved with Priority Review and Breakthrough Therapy protocols.


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