title: FDA-Approved Oligonucleotide Therapies in 2017.
abstract: 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.
authors.affiliation: Department of Medical Oncology and Experimental Therapeutics, City of Hope Medical Center, 1500 East Duarte Road, Duarte, CA 91010, USA. Electronic address: cstein@coh.org.
authors.forename: Cy A
authors.initials: CA
authors.lastname: Stein
authors.affiliation: Department of Medical Oncology and Experimental Therapeutics, City of Hope Medical Center, 1500 East Duarte Road, Duarte, CA 91010, USA.
authors.forename: Daniela
authors.initials: D
authors.lastname: Castanotto
mesh_terms: D052157:Aptamers, Nucleotide / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D017726:Cytomegalovirus Retinitis / Q000235:genetics / Q000628:therapy / Q000821:virology; D017277:Drug Approval; D006504:Hepatic Veno-Occlusive Disease / Q000235:genetics / Q000473:pathology / Q000628:therapy; D006801:Humans; D006937:Hypercholesterolemia / Q000235:genetics / Q000473:pathology / Q000628:therapy; D008268:Macular Degeneration / Q000235:genetics / Q000473:pathology / Q000628:therapy; D060172:Morpholinos / Q000627:therapeutic use*; D009134:Muscular Atrophy, Spinal / Q000235:genetics / Q000473:pathology / Q000628:therapy; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000473:pathology / Q000628:therapy; D009841:Oligonucleotides / Q000627:therapeutic use*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*; D011089:Polydeoxyribonucleotides / Q000627:therapeutic use*; D013873:Thionucleotides / Q000627:therapeutic use*
chemical_list: D052157:Aptamers, Nucleotide; D060172:Morpholinos; D009841:Oligonucleotides; D016376:Oligonucleotides, Antisense; D011089:Polydeoxyribonucleotides; D013873:Thionucleotides; C495058:pegaptanib; C036901:defibrotide; C000590926:nusinersen; C524142:mipomersen; C000611335:eteplirsen; C091346:fomivirsen
keywords: Defitelio; Eteplirsen; Kynamro; Macugen; Spinraza; Vitravene; aptamer; oligonucleotide; phosphorothioate; therapeutics