Exondys 51: how long were the pivotal tox studies?

“39-Week Intravenous Injection Toxicity and Toxicokinetic Study of Eteplirsen in Sexually Mature Male Cynomolgus Monkeys with an 8-Week Recovery”

“Pivotal toxicology studies of eteplirsen were conducted in male monkeys (39-week study) and juvenile male rats (10-week study). A 26-week study was conducted with a mouse-specific surrogate (AVI-4225) in male transgenic mdx mice.”

“In the pivotal studies, eteplirsen was administered weekly at doses of 0, 5, 40, and 320 mg/kg IV; the 12-week study also included a group receiving 320 mg/kg SC.”