Per CardioBrief " Because of their small size the mipomersen trials were not powered to #$%$ cardiovascular outcomes, though cardiovascular benefit is of course the ultimate intended effect of the drug. However, the numbers in the phase 3 studies ran in the wrong direction. Serious adverse events of cardiac disorders occurred in 3.8% (10/261) of patients in the mipomersen group compared with 3.1% (4/129) in the placebo group. The reviewers concluded that “the possibility that mipomersen therapy increases the risk for cardiovascular events cannot be excluded.”
These are not children just diagnosed. These are lifetime severe FH. That conclusion is utterly wrong, given the numbers. 50% die from cardio events by age 50. That IS the disease indication. Do that math. If you watch the numbers carefully, it not out of line at all. 8 cardio events would have been normal. How many in the non-placebo group also took statins at the same time??? Even if only 6 had events in the non-placebo group the general statement issues still applies. Even if NONE had a cardio event, the same general statement applies. All it implies is over time, when Kynamro can be used earlier, in patients with less exposure to statins and in younger settings, maybe the very opposite can be proven.
Remember who ISIS partners are and how they were involved in the trial designs. They know more than the FDA. Much more. Sanofi certainly knows if, how and when a given drug ties to cancer in a trial setting.