Ionis Pharmaceuticals Inc. and its affiliate Akcea Therapeutics Inc. said their drug Waylivra helped patients with a rare lipid disorder control fat levels in a late-stage study.
Familial partial lipodystrophy, or FPL, which is characterized by abnormal fat distribution, leads to high levels of fat in the liver and muscle, as well as the bloodstream. Patients with the disease can also show a host of metabolic abnormalities, and the conditions can lead to problems with other organs, as well, leading to serious complications, according to Ionis and Akcea.
In initial results from the phase 3 study called BROADEN, patients with FPL showed a significant reduction in fat levels compared to patients taking a placebo. The patients showed 88% reduction in triglyceride levels, which is fat stored in the blood.
Waylivra, which is an antisense drug that targets genetic material to interrupt disease-causing protein creation, reached its secondary goal of reducing liver fat by more than half.
Waylivra gained European approval in May for another lipid disorder called familial chylomicronemia syndrome. The U.S. Food and Drug Administration had previously rejected the drug in August 2018.