Qalsody: how long were the pivotal tox studies?

“Repeat IT dosing with 0, 4, 12, and 35 mg tofersen every 2 weeks for a 9-month period resulted in uncoordinated leg movement and transient impairments of patellar and foot-grip reflexes in HDM and HDF.”

“Nonclinical toxicity studies submitted to the NDA consisted of single dose (rat) and repeat dose (mouse and monkey) general toxicity studies, as well a complete battery of genetic toxicology and reproductive and developmental toxicology studies.”

“SC administration of 0, 6, 30, and 150 mg/kg tofersen every other week for 26 weeks in male and female CD1 mice resulted in dose-dependent reductions in platelet counts in males (up to 20%) and in HDF (13%).”