Spinraza: how long were the pivotal tox studies?

“A one year (53 weeks) repeat dose toxicity study in juvenile cynomolgus monkeys with a 26-week recovery phase”

“The toxicity and pharmacokinetics (PK) of ISIS 396443 were evaluated following single IT bolus doses (up to 7 mg) in adult monkeys or following repeated IT bolus doses (weekly or every other week) for 14 (0.3, 1, or 3 mg/dose) and 53 (0.3, 1, or 4 mg/dose) weeks in juvenile monkeys.”

“CNS safety was assessed as part of the acute and subchronic toxicity studies in monkeys using IT lumbar bolus injection, the intended route of administration in man.”