FDA News

FDA approves pexidartinib, first therapy for tenosynovial giant cell tumor

The FDA announced today that it approved pexidartinib for the treatment of adults with symptomatic tenosynovial giant cell tumor that is linked to functional limitations or severe morbidity and does not improve with surgery.

The drug (Turalio, Daiichi Sankyo) is the first therapy for this rare joint tumor, according to a press release from the FDA.

“TGCT (tenosynovial giant cell tumor) can cause debilitating symptoms for patients such as pain, stiffness and limitation of movement,” Richard Pazdur, MD, director of the Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products at the Center for Drug Evaluation and Research for the FDA, said in the release. “The tumor can significantly affect a patient’s quality of life and cause severe disability. Surgery is the primary treatment option, but some patients are not eligible for surgery, and tumors can recur even after the procedure.”

The agency’s approval was based on results of the ENLIVEN trial. As HemOnc Today previously reported, results presented at the ASCO Annual Meeting in 2018 showed 39% of patients assigned pexidartinib responded to treatment compared with 0% of patients assigned placebo (P < .0001).

The prescribing information for the therapy includes a boxed warning about the risk for serious and potentially fatal liver injury, according to the release. Liver tests should be monitored before and during treatment.

The drug is currently only available through the Risk Evaluation and Mitigation Strategy Program, according to the release.

Disclosure: Pazdur is an employee of the FDA.

 

The FDA announced today that it approved pexidartinib for the treatment of adults with symptomatic tenosynovial giant cell tumor that is linked to functional limitations or severe morbidity and does not improve with surgery.

The drug (Turalio, Daiichi Sankyo) is the first therapy for this rare joint tumor, according to a press release from the FDA.

“TGCT (tenosynovial giant cell tumor) can cause debilitating symptoms for patients such as pain, stiffness and limitation of movement,” Richard Pazdur, MD, director of the Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products at the Center for Drug Evaluation and Research for the FDA, said in the release. “The tumor can significantly affect a patient’s quality of life and cause severe disability. Surgery is the primary treatment option, but some patients are not eligible for surgery, and tumors can recur even after the procedure.”

The agency’s approval was based on results of the ENLIVEN trial. As HemOnc Today previously reported, results presented at the ASCO Annual Meeting in 2018 showed 39% of patients assigned pexidartinib responded to treatment compared with 0% of patients assigned placebo (P < .0001).

The prescribing information for the therapy includes a boxed warning about the risk for serious and potentially fatal liver injury, according to the release. Liver tests should be monitored before and during treatment.

The drug is currently only available through the Risk Evaluation and Mitigation Strategy Program, according to the release.

Disclosure: Pazdur is an employee of the FDA.