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

“Vamorolone was negative in an adequately conducted battery of genotoxicity studies that consisted of an in vitro bacterial reverse mutation assay, an in vitro mammalian gene mutation assay in mouse lymphoma cells, and an in vivo micronucleus assay in mouse.”

“given the seriousness of the indication, carcinogenicity studies in two species (mouse and rat) are to be conducted as post marketing requirements.”

“Because the intended patient population is almost exclusively male, a standard battery of reproductive and developmental toxicology studies was not needed to support a marketing application”

“Vamorolone did not inhibit human ether-a-go-go-related gene (hERG) potassium currents up to the highest concentration of 20 µM in vitro. Accordingly, oral vamorolone doses up to 50 mg/kg did not impair cardiovascular and respiratory function in telemetered dogs.”