Vitravene |
Fomivirsen |
1998 |
Antisense oligonucleotide |
Cytomegalovirus retinitis |
Intravitreal injection |
None |
Isis Pharmaceuticals |
Macugen |
Pegatanib |
2004 |
Aptamer |
Age-related macular degeneration |
Intravitreal injection |
None |
Eyetech Inc. |
Kynamro |
Mipomersen |
2013 |
Antisense oligonucleotide |
Homozygous familial
hypercholesterolemia |
Subcutaneous injection |
None |
Genzyme Corporation |
Exondys 51 |
Eteplirsen |
2016 |
Antisense oligonucleotide |
Duchenne muscular dystrophy |
Intravenous infusion |
None |
Sarepta Therapeutics |
Spinraza |
Nusinersen |
2016 |
Antisense oligonucleotide |
Spinal muscular atrophy |
Intrathecal injection |
None |
Biogen Inc. |
Defitelio |
Defibrotide |
2016 |
Oligonucleotide with profibrinolytic
properties |
Sinusoidal obstruction syndrome |
Intravenous infusion |
None |
Jazz Pharmaceuticals, Inc. |
Onpattro |
Patisiran |
2018 |
Small interference RNA |
Polyneuropathy of hereditary transthyretin-mediated
amyloidosis |
Intravenous infusion |
Lipid complex |
Alnylam Pharma Inc. |