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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 

Reference http://www.medscape.com/viewarticle/778465  

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