Date: 2015-08-03
Type of information: Results
phase: 3
Announcement: results
Company: Isis Pharmaceuticals (USA - CA)
Product: Kynamro® (mipomersen sodium)
Action
mechanism: antisense oligonucleotide. Mipomersen is a first-in-class apo-B synthesis inhibitor and acts by blocking the production of apolipoprotein B (apoB), the protein that provides the structural core for these atherogenic particles, including LDL and lipoprotein-a (Lp(a)).
Disease: severe heterozygous familial hypercholesterolemia (severe HeFH)
Therapeutic area: Cardiovascular diseases - Genetic diseases
Country: Argentina, Australia, Belgium, Brazil, Canada, Croatia, Czech Republic, Denmark, Germany, Greece, Hungary, India, Israel, Italy, Republic of Korea, Malaysia, The Netherlands, New Zealand, Norway, Russian Federation, South Africa, Spain, Sweden, Taiwan, Turkey, Ukraine, UK, USA
Trial
details: FOCUS FH was a multicenter, randomized, placebo-controlled, double-blind, parallel-group study that enrolled 310 patients aged 18 and older, followed by an open-label continuation. Cohort 1 included patients with severe HeFH with LDL-C ≥ 200 mg/dL plus coronary heart disease or LDL-C ≥ 300 mg/dL. Cohort 2 included patients with HeFH with LDL-C ≥ 160 mg/dL and <200 mg/dL plus coronary heart disease. Within each cohort, patients were randomized 2:1 to either 200 mg once weekly, 70 mg thrice weekly, or placebo for a 60 week study duration. Upon completion of the 60 week blinded treatment, patients had the option to enter the open label continuation period for 26 weeks and receive the full dose regimen of KYNAMRO according to the dosing schedule they were randomized to during the blinded treatment phase. The trial was conducted at 131 sites worldwide. The primary efficacy endpoint evaluated was the LDL-C percent change from baseline to week 61 for cohort 1 and each dose regimen. (NCT01475825)
Latest
news: * On August 3, 2015, Isis Pharmaceuticals announced that the FOCUS FH phase 3 study of Kynamro® (mipomersen sodium) in patients with severe heterozygous familial hypercholesterolemia (severe HeFH) met its primary efficacy endpoint, a statistically significant reduction in LDL-cholesterol after 60 weeks of treatment of once weekly injections of 200 mg of Kynamro® compared to placebo. LDL-cholesterol reduction was similar to that observed in previous phase 3 studies. In addition, based on the data available for review, the safety profile of Kynamro® observed in the FOCUS FH trial was similar to the safety profile reported in previous phase 3 studies. Genzyme will provide a more in depth review of the safety and efficacy data at a future medical meeting.
