Thursday's panel will only review mipomersen as a treatment for HoFH. In a phase III study, 34 patients treated with mipomersen for 26 weeks saw 25% reduction in LDL cholesterol compared to a 3% reduction in patients treated with a placebo.
Like with lomitapide, fatty liver and elevated liver enzyme levels were worrisome side effects recorded in the mipomersen phase III trial. Injection site reactions were also reported by 76% of mipomersen patients.
-- Reported by Adam Feuerstein in Boston.