TABLE 3.
U.S. FDA Novel Orphan Drug Approvals in 2020–2021
Only those drugs considered first-in-class are shown: 26 of 50 (52%). Some 21 of those drugs (81%) reflected first-in-class approvals, an indication of the strong innovation. 2020: CDER approved 53 novel drugs, 31 orphan drugs (58%) (Source: FDA; taken from https://globalgenes.org/raredaily/a-group-of-innovative-orphan-drugs-win-fda-approval-in-2021/?gclid=CjwKCAiAyfybBhBKEiwAgtB7fmCXF8VAW0aHnwGHogVoFWwiRqkm GXkSPD-dZLyIaB6QXdFwwrw-FBoCMVcQAvD_BwE).
| Proprietary Name | Active Ingredient | Indication | |
|---|---|---|---|
| Besremi | Ropeginterferon alfa-2b-njft | To treat polycythemia vera | |
| Bylvay | Odevixibat | To treat pruritus in progressive familial intrahepatic cholestasis |
|
| Cytalux | Pafolacianine | To use for ovarian cancer imaging | |
| Empaveli | Pegcetacoplan | To treat paroxysmal nocturnal hemoglobinuria | |
| Evkeeza | Evinacumab-dgnb | To treat homozygous familial hypercholesterolemia | |
| Livtencity | Maribavir | To treat cytomegalovirus infection | |
| Lumakras | Sotorasib | To treat types of non–small cell lung cancer | |
| Nulibry | Fosdenopterin | To reduce the risk of mortality in molybdenum cofactor deficiency type A | |
| Rezurock | Belumosudil | To treat chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy | |
| Tavneos | Avacopan | To treat severe active antineutrophil cytoplasmic autoantibody-associated vasculitis in combination with standard therapy, including glucocorticoids | |
| Voxzogo | Vosoritide | To improve growth in children 5 years of age and older with achondroplasia and open epiphyses |
|
| Vyvgart | Efgartigimod alfa-fcab | To treat generalized myasthenia gravis | |
| Welireg | Belzutifan | To treat von Hippel-Lindau disease | |
| Zynlonta | Loncastuximab tesirine-lpyl | To treat types of relapsed or refractory large B-cell lymphoma |