Table 5. Therapeutic proteins granted orphan designation upon original submission (2011–2016).
| # | Approval
date |
Drug name
(Market name) |
Class | Description | Orphan designation |
|---|---|---|---|---|---|
| 1 | 3/25/2011 | Ipilimumab
(Yervoy) |
mAb | Human anti-CTLA-4 | Treatment of high-risk stage II, stage III,
and stage IV melanoma |
| 2 | 6/15/2011 | Belatacept
(Nulojix) |
Fusion (Fc) | CTLA-4 Fc-fusion | Prophylaxis of organ rejection in renal
allograft recipients |
| 3 | 11/18/2011 | Asparaginase erwinia
chrysanthemi (Erwinaze) |
Enzyme | Asparaginase erwinia
chrysanthemi |
Treatment of acute lymphocytic
leukemia |
| 4 | 1/17/2012 | Glucarpidase
(Voraxaze) |
Enzyme | Glucarpidase | Treatment of patients at risk of
methotrexate toxicity |
| 5 | 5/1/2012 | Taliglucerase alfa
(Elelyso) |
Enzyme | Taliglucerase | Treatment of Gaucher’s disease |
| 6 | 12/14/2012 | Raxibacumab
(raxibacumab) |
mAb | Human anti-anthrax
protective antigen (PA) |
Treatment of anthrax |
| 7 | 6/26/2013 | Coagulation factor IX
recombinant human (Rixubis) |
Coagulation factor | Recombinant factor IX | Prophylactic use to prevent or reduce
the frequency of bleeding episodes in patients with hemophilia B (routine prophylaxis in patients where there is no evidence or suspicion of bleeding) |
| 8 | 11/1/2013 | Obinutuzumab
(Gazyva) |
mAb | Humanized anti-CD20 | Treatment of chronic lymphocytic
leukemia |
| 9 | 12/23/2013 | Coagulation factor
XIII A-subunit (recombinant) (Tretten) |
Coagulation factor | Recombinant factor XIII
A subunit |
Prophylaxis of bleeding associated with
congential factor XIII deficiency |
| 10 | 2/14/2014 | Elosulfase alfa
(Vimizim) |
Enzyme | Elosulfase alfa | Treatment of mucopolysaccharidosis
type IV A (Morquio A syndrome) |
| 11 | 2/24/2014 | Metreleptin
(Myalept) |
Hormone | Metreleptin | Treatment of metabolic disorders
secondary to lipodystrophy |
| 12 | 3/28/2014 | Coagulation factor IX
(recombinant), Fc fusion protein (Alprolix) |
Coagulation factor | Recombinant factor IX
Fc fusion |
Control and prevention of hemorrhagic
episodes in patients with hemophilia B (congenital factor IX deficiency or Christmas disease) |
| 13 | 4/21/2014 | Ramucirumab
(Cyramza) |
mAb | Human anti-VEGFR2
(KDR) |
Treatment of gastric cancer |
| 14 | 4/23/2014 | Siltuximab (Sylvant) | mAb | Mouse/human chimeric
anti-IL-6 |
Treatment of Castleman’s disease |
| 15 | 4/23/2014 | Pembrolizumab
(Keytruda) |
mAb | Humanized anti-PD-1 | Treatment of stage IIB through IV
malignant melanoma |
| 16 | 6/6/2014 | Antihemophilic
factor (recombinant), Fc fusion protein (Eloctate) |
Coagulation factor | Recombinant factor VIII
Fc-fusion |
Treatment of hemophilia A |
| 17 | 7/16/2014 | C1 esterase inhibitor
recombinant (Ruconest) |
Plasma protein | Recombinant C1
esterase inhibitor |
Treatment of (acute attacks of)
angioedema caused by hereditary or acquired C1-esterase inhibitor deficiency |
| 18 | 10/23/2014 | Antihemophilic factor
porcine, B-domain truncated recombinant (Obizur) |
Coagulation factor | Recombinant factor VIII
(porcine) |
Treatment and prevention of episodic
bleeding in patients with inhibitor antibodies to human coagulation factor VIII |
| 19 | 12/3/2014 | Blinatumomab
(Blincyto) |
mAb | Mouse bispecific anti-
CD19/anti-CD3 |
Treatment of acute lymphocytic
leukemia |
| 20 | 12/22/2014 | Nivolumab
(Opdivo) |
mAb | Human anti-PD-1 | Treatment of stage IIb to IV melanoma |
| 21 | 1/23/2015 | Parathyroid hormone
(Natpara) |
Hormone | Parathyroid hormone | Treatment of hypoparathyroidism |
| 22 | 3/10/2015 | Dinutuximab
(Unituxin) |
mAb | Mouse/human chimeric
anti-GD2 |
Treatment of neuroblastoma |
| 23 | 8/27/2015 | Evolocumab
(Repatha) |
mAb | Human anti-proprotein
convertase substilisin/ kexin type 9 (PCSK9) |
Treatment of homozygous familial
hypercholesterolemia |
| 24 | 10/16/2015 | Idarucizumab
(Praxbind) |
Fab | Humanized anti-
dabigatran |
To reverse the anticoagulant effect of
dabigatran due to uncontrolled life- threatening bleeding requiring urgent intervention or a need to undergo an emergency surgery/urgent invasive procedure |
| 25 | 10/23/2015 | Asfotase-alfa
(Strensiq) |
Enzyme/fusion
protein |
Tissue non-specific
alkaline phosphatase/ Fc fusion/deca- asparatate (D10) peptide |
Treatment of hypophosphatasia |
| 26 | 11/16/2015 | Daratumumab
(Darzalex) |
mAb | Human anti-CD38 | Treatment of multiple myeloma |
| 27 | 11/24/2015 | Necitumumab
(Portrazza) |
mAb | Human anti-epidermal
growth factor receptor |
Treatment of squamous non-small cell
lung cancer |
| 28 | 12/8/2015 | Sebelipase alfa
(Kanuma) |
Enzyme | Lysosomal acid lipase | Treatment of lysosomal acid lipase
deficiency |
| 29 | 12/8/2015 | von Willebrand Factor
(Recombinant) (Vonvendi) |
Plasma protein | Recombinant von
Willebrand Factor |
Treatment of von Willebrand disease |
| 30 | 3/4/2016 | Coagulation factor IX
recombinant human (Idelvion) |
Coagulation factor | Recombinant factor IX
albumin fusion |
Treatment of patients with congenital
factor IX deficiency (hemophilia B) |
| 31 | 3/18/2016 | Obiltoxaximab
(Anthim) |
mAb | Mouse/human chimeric
anti- Bacillus anthracis |
Treatment of exposure to
B. anthracis
spores |
Comprehensive listing of all FDA-approved therapeutic proteins granted orphan designation upon original submission from January 1, 2011, through August 31, 2016, listed in chronological order of FDA approval. In addition, the class of protein, a brief description, and orphan designation are included. CD, cluster of differentiation; CTLA-4, cytotoxic T-lymphocyte-associated protein 4; Fab, fragment antigen binding; Fc, fragment crystallizable; GD2, disialoganglioside; IL, interleukin; mAb, monoclonal antibody; PD-1, programmed death receptor-1; VEGFR, vascular endothelial growth factor receptor.