Ryoncil: what was done before first-in-human, and what came after?

“No formal pharmacokinetic, clinical dose finding, or dose regimen optimization studies have been performed during the development of RYONCIL for treatment of aGvHD in pediatrics.”

“In a 90-day GLP repeat dose toxicity study, rats received 2×106, 10×106 or 20×106 rat ceMSC/kg administered in a 5mL/kg dosing volume by tail vein injection over a two-minute period twice per week for 4 weeks and weekly thereafter for a total of 13 dose administrations.”

“In a GLP repeat dose toxicity study, rats received 2x106 , 10x106 or 20x106 rat ceMSC/kg administered in a 5mL/kg dosing volume by tail vein injection over a two-minute period”

“Genotoxicity, carcinogenicity and reproductive and developmental toxicity studies were not performed for remestemcel-L. These studies are not warranted based on the product characteristics and safety profile.”

“Genotoxicity, carcinogenicity and reproductive and developmental toxicity studies were not performed for remestemcel-L.”

“Genotoxicity, carcinogenicity, and reproductive and developmental toxicity studies were not performed for RYONCIL. These studies are not warranted based on the product characteristics and safety profile.”

“In a tumorigenicity study in athymic mice, administration of human ceMSC from five separate donors was evaluated at dose levels up to 50x106 cells/kg. There was no evidence of neoplastic tissue formation attributable to ceMSC over the six-week duration of the study.”

“In a tumorigenicity study in athymic mice, administration of human ceMSC from five separate donors was evaluated at dose levels up to 50x106 cells/kg. There was no evidence of neoplastic tissue formation attributable to ceMSC over the six-week duration of the study.”

“Genotoxicity, carcinogenicity, and reproductive and developmental toxicity studies were not performed for RYONCIL. These studies are not warranted based on the product characteristics and safety profile.”