Mipomersen Is Well Tolerated, Maintains Activity In FH Patients
Isis Pharmaceuticals announced additional results from its ongoing Phase 2 open-label extension (OLE) study of mipomersen in patients with familial hypercholesterolemia (FH). In FH patients exposed to mipomersen for as long as 16 months, the safety profile of the drug continues to be encouraging. Reductions in LDL-cholesterol were also maintained in patients who had responded to treatment in their initial study and continued on treatment.
In this OLE study, patients with FH who were initially enrolled in one of two Phase 2 efficacy studies of mipomersen were allowed to enter the OLE study. All patients in the study were being treated with maximum-tolerated lipid-lowering therapy before and during the study. Most patients received 200 mg/week of mipomersen in addition to maximum-tolerated lipid-lowering therapy. The median duration of continuous exposure in these patients was 38 weeks. The maximum continuous exposure was 64 weeks.
All patients in the study had the opportunity to self-administer mipomersen. Although the study was not designed to measure efficacy, the activity of mipomersen was clearly demonstrated and appeared to be well maintained with longer-term dosing.
With respect to safety, the drug was well tolerated in longer-term treatment. There were no new types of adverse events observed and rates of adverse events decreased during the OLE study from those in the initial efficacy studies.
The following additional observations were made in these patients: