The indication is for symptomatic tenosynovial giant cell tumor TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity. The FDA approved vimseltinib Deciphera Pharmaceuticals for the treatment of adult patients with symptomatic tenosynovial giant cell tumor TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity.
Developed using a proprietary switch-control kinase inhibitor platform, vimseltinib is an investigational oral switch-control tyrosine kinase inhibitor designed to selectively inhibit colony-stimulating factor 1 receptor CSF1R.
Although these tumors are benign, they can grow and cause damage to surrounding tissues and structures, inducing pain, swelling, and limitation of movement of the joint. Surgery is the primary treatment option; however, the tumors often recur—particularly in diffuse-type TGCT—and if left untreated or if it continually recurs, damage and degeneration may develop in the affected joint and surrounding tissues, potentially causing significant disability.
In part 2, patients who received placebo in part 1 were given the option to receive vimseltinib and received the drug for a long-term period in an open-label setting. The primary end point was objective response rate ORR in the intent to treat population compared with placebo, and secondary end points include ORR per tumor volume score, active range of motion, physical function, stiffness, quality of life, and pain, all of which were assessed at 25 weeks day 1 of cycle 7.
Most of the treatment-emergent adverse events AEs observed were grades 1 or 2 in severity. There was 1 incidence of serious subcutaneous abscess, and no evidence of cholestatic hepatotoxicity or drug-induced liver injury was observed.
