Ionis Pharmaceuticals’ olezarsen has Phase 3 results showing the therapy handily beat a placebo at reducing fat levels in the blood due to a rare, inherited metabolic disorder with no FDA-approved drugs. Ionis plans early 2024 submissions for what could become the first medicine it commercializes without a partner.

An analyst wrote that while the data are positive, the bigger question is how the drug will be priced, based on another drug already on the market for the same disease, HoFH, which affects about 1,300 people in the U.S.

The study out of Vanderbilt University Medical Center found 14 percent of individuals with genetic variants impacting the kidney had kidney transplants and 10 percent with another variant had liver transplants. These transplants could have been avoided had the underlying genetic cause been disclosed.