nusinersen and defibrotide

Oligonucleotides (oligos) have been under clinical development for approximately the past 30 years, beginning with antisense oligonucleotides (ASOs) and apatmers and followed about 15 years ago by siRNAs. During that lengthy period of time, numerous clinical trials have been performed and thousands of trial participants accrued onto studies. Of all the molecules evaluated as of January 2017, the regulatory authorities assessed that six provided clear clinical benefit in rigorously controlled trials. The story of these six is given in this review.

Topics: Aptamers, Nucleotide; Clinical Trials as Topic; Cytomegalovirus Retinitis; Drug Approval; Hepatic Veno-Occlusive Disease; Humans; Hypercholesterolemia; Macular Degeneration; Morpholinos; Muscular Atrophy, Spinal; Muscular Dystrophy, Duchenne; Oligonucleotides; Oligonucleotides, Antisense; Polydeoxyribonucleotides; Thionucleotides

Topics: Acetamides; Antibodies, Monoclonal; Antibodies, Monoclonal, Humanized; Antitoxins; Bridged Bicyclo Compounds, Heterocyclic; Carbazoles; Chenodeoxycholic Acid; Drug Approval; Humans; Indoles; Morpholinos; Oligonucleotides; Piperidines; Polydeoxyribonucleotides; Pyrazines; Sulfonamides; United States; United States Food and Drug Administration