Exposure-response analyses for belzutifan to inform dosing considerations and labeling

Two clinical studies looked at the safety and efficacy of belzutifan, an oral, potent hypoxia-inducible factor-2α inhibitor, recently approved in the United States for the treatment of von Hippel–Lindau (VHL) disease-associated RCC and other VHL disease-associated neoplasms. Among other things, if was determined that the efficacy exposure-response (E-R) was generally flat with non-significant positive trends with exposure, and the safety E-R analyses demonstrated a lack of relationship for grade ≥3 hypoxia and a positive relationship for grade ≥3 anemia, with incidences also significantly dependent on baseline Hgb.