Fabhalta: genotox, carcinogenicity and repro: done or skipped?

“Iptacopan was not genotoxic or mutagenic in a battery of in vitro and in vivo assays.”

“Carcinogenicity studies conducted with oral administration of iptacopan in RasH2 transgenic mice with doses up to 1,000 mg/kg/day for 6 months and in rats with doses up to 750 mg/kg/day for 2 years did not identify any carcinogenic potential.”

“In animal reproduction studies, oral administration of iptacopan to pregnant rats and rabbits during organogenesis at exposures 4- to 6-times the human exposure (based on AUC) at the maximum recommended human dose (MRHD) of 200 mg twice daily did not induce embryo or fetal toxicity (see Data) .”

“Iptacopan-related decrease in blood pressure up to 26 mmHg (23%) and a concurrent increase in heart rate up to 40 beats/min (43%) compared to baseline-adjusted values in the control group.”