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

“Therefore, the carcinogenicity potential for pegaptanib sodium is considered low, based on the negative in vitro and in vivo genotoxicity assays for pegaptanib sodium and its component modified nucleotides.”

“The main argument considered by the CHMP in order to accept the lack of carcinogenicity studies was due to the very low systemic exposure obtained in the clinical population together with the apparent difficulty to perform relevant study obtaining both high systemic and intraocular exposure.”

“The reproductive toxicology program included range-finding embryo-foetal development studies in rabbit and mouse and a final embryo-foetal development study in the mouse.”

“Pegaptanib sodium was evaluated in animal models for cardiovascular, respiratory and neurobehavioral effects, at IV administered doses with associated systemic exposures up to 10-fold”