A therapeutic antisense oligonucleotide inhibitor of apolipoprotein B mRNA.
It made it past Phase 3 studies and was approved by the FDA* in 2013 for managing the über rare–i.e., orphan disease homozygous familial hypercholesterolaemia–FH.
Adverse effects Angina, palpitations, malignancy, immune-mediated reactions, hepatic changes including toxicity
*The FDA voted 9 to 6 for approval. The European Medicines Agency recommended against approving mipomersen for FH.
Alternative names ISIS 301012, Kynamro