Regorafenib for patients with KIT-mutant advanced melanoma

Oncogenic KIT-mutations are found in ~3% of melanoma patients. While only a minority of these patients respond to a treatment with small molecule KIT-inhibitors, such as imatinib, sunitinib, or nilotinib, more promising activity has been seen with the multi-targeted small molecule inhibitor regorafenib. During ESMO 2024, Dr Iris Dirven, medical oncologist in training at the University Hospital Brussels, shared the results obtained with regorafenib in 8 KIT-mutant advanced melanoma patients who exhausted all the available treatment option.

After a median time on regorafenib of 37 weeks, an objective response rate of 100% was reported, with a median duration of response of 37.4 weeks (ongoing responses in 4 patients). Furthermore, continuous dosing of regorafenib 40-80 mg once daily proved to be associated with a manageable safety profile.

As such, regorafenib demonstrates high anti-tumor activity in this Caucasian population of heavily pre-treated, KIT-mutant advanced melanoma patients warranting further prospective evaluation.