Trikafta: how long were the pivotal tox studies?

“When VX-445 was administered alone chronically (up to 39-week) in dogs (VX-445-TX-006), test item-related microscopic changes were noted at 14 mg/kg/day including hepatocellular hypertrophy, epithelium vacuolation in gallbladder, and testicular seminiferous tubule atrophy/degeneration.”

“As listed in the following table, general toxicity studies with either VX-445 alone or in combination with tezacaftor (VX-661) and ivacaftor (VX-770) were conducted in rats, mice, and dogs with dosing durations up to 6 months in rodents and 9 months in non-rodents.”

“VX-445: A 26-week Oral (Gavage) Toxicity Study in Sprague Dawley Rats Followed by a 6-week Recovery Period”