The U.S. Food and Drug Administration approved Japanese drugmaker Daiichi Sankyo Co. Ltd.'s Turalio as the first treatment for a rare tumor.
The company's Turalio, or pexidartinib, capsules were cleared to treat adults with symptomatic tenosynovial giant cell tumor — a rare, usually, noncancerous tumor that affects the synovium-lined joints, bursae and tendon sheaths. The condition results in swelling, pain, stiffness and reduced mobility in the affected joint or limb.
Turalio becomes the first approved therapy in the U.S. for this type of tumor, the primary treatment option for which has been surgery, the agency said. However, some patients are not eligible for surgery and tumors can recur even after the procedure.
The approval was based on data from a late-stage study, which showed that the medicine helped reduce tumor size in patients.
The U.S. regulator was undertaking a priority review of the company's application for Turalio, which was also granted the breakthrough therapy designation orphan-drug status.
The label for Turalio includes a boxed warning flagging the risk of serious and potentially fatal liver injury. In October 2016, Daiichi discontinued enrollment in its Enliven study evaluating Turalio after reviewing two cases of nonfatal and serious liver toxicity.