Oligonucleotides, Antisense

Short fragments of DNA or RNA that are used to alter the function of target RNAs or DNAs to which they hybridize.
DrugDrug NameDrug Indication
DB05528MipomersenUsed in patients with homozygous familial hypercholesterolemia as an adjunct to diet and other lipid-lowering medications.
DB05487CustirsenInvestigated for use/treatment in brain cancer and breast cancer.
DB06759FomivirsenIndicated for the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency syndrome (AIDS), when other therapy has been ineffective or is considered unsuitable [FDA Label, L1428].
DB13811OblimersenNot Available
DB06014EteplirsenEteplirsen is indicated for treatment of certain individuals with Duchenne muscular dystrophy (DMD). Its use is limited to those with a confirmed mutation of the DMD gene which would benefit from exon 51 skipping. Based on clinical studies showing increased dystrophin production in skeletal muscle in some patients given this drug, the above indication was approved under accelerated approval. Further confirmatory trials are required to demonstrate clinical benefit of eteplirsen.
DB13161NusinersenIndicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
DB14713InotersenInotersen is a transthyretin-directed antisense oligonucleotide indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults [FDA label], [F1938].