Table 3 -. Approved products for gene silencing.
| Type | Name | Indication | Method* | Immune-related issues** | Situation |
|---|---|---|---|---|---|
| in vivo | |||||
| Vitravene (fomivirsen) | Cytomegalovirus retinitis | Gene-silencing antisense therapy (ASO - antisense oligonucleotide) | Uveitis, including iritis and vitritis. Conjunctival and retinal inflammation. Anaemia, asthenia, diarrhea, fever, infection, rash, sepsis. More rare: abnormal liver function, allergic reactions, kidney failure, lymphoma like reaction, neuropathy, neutropenia, pancreatitis, thrombocytopenia. | 2002 - Withdrawn (EU) 2006 - Withdrawn (US) | |
| Macugen (pegaptanib) | Neovascular age-related macular degeneration (AMD) | RNA oligonucleotide | Anterior chamber inflammation, punctate keratitis. Rare cases of anaphylaxis/anaphylactoid reactions, including angioedema, have been reported. | 2004 - Approved (FDA) 2011 - Withdrawn (EU) | |
| Kynamro (mipomersen sodium) | Homozygous familial hypercholesterolemia (HoFH) | Gene-silencing antisense therapy (ASO) | Elevation of alanine aminotransferase, Hepatic steatosis, influenza-like illness, pyrexia, arthralgia. | 2013 - Approved | |
| Exondys 51 (eterplisen) | Duchenne muscular distrophy | ASO | Contact dermatitis | 2016 - Approved | |
| Spinraza (nusinersen) | Spinal muscular atrophy (SMA) | Gene-silencing antisense therapy (ASO) | Thrombocytopenia and Coagulation Abnormalities, Renal Toxicity, lower respiratory infection and upper respiratory infection. | 2016 - Approved | |
| Tegsedi (inotersen) | Transthyretin-mediated amyloidosis (hATTR) | ASO | Injection site reactions and fever. Thrombocytopenia, glomerulonephritis, renal toxicity, hepatic dysfunction, strokes. Rarer: antineutrophil cytoplasmic autoantibody (ANCA)-positive systemic vasculitis. | 2018 - Approved | |
| Patisiran (onpattro) | Polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) | RNA interference | Upper respiratory tract infections and infusion-related reactions. | 2018 - Approved | |
| Vyondis 53 (golodirsen) | Duchenne muscular distrophy | ASO | Hypersensitivity reaction, rash, pyrexia, pruritus, urticaria, dermatitis, skin exfoliation, nasopharyngitis. Renal toxicity. | 2019 - Approved | |
| Waylivra (volanesorsen) | Lipoprotein lipase deficiency; Hypertriglyceridaemia | ASO | Injection site reactions. Allergic reaction including rash and fever. Thrombocytopenia, renal and hepatic toxicity. | 2019 - Approved |
* As described in the approval document. ** Information obtained from package insert, unless indicated otherwise.