Glybera: how long were the pivotal tox studies?

“Moreover, the long-term expression of the therapeutic gene has been taken into account by including an extended follow-up period of 6 months in one of the single dose toxicity studies performed with AMT-011.”

“The non-clinical toxicology programme of Glybera included one single dose toxicity study performed with AMT-010 with a 90-day observation period, two single dose toxicity studies performed with AMT-011 with a 180-day and 105-day observation period, respectively, and a combined reproductive toxicology and breeding study performed with AMT-011.”

“The single dose toxicity studies reflect the clinical dosing situation by addressing the toxic effects following either IM (clinical use) or IV (worst case scenario) administration of AMT-011 at a single occasion.”