TABLE 1.
Pediatric Orphan Indications for Novel Drugs, 2010–2018 (N = 60)
| Generic Name | Disease Population(s) | Generic Name | Disease Population(s) |
|---|---|---|---|
| Antihemophilic factor (recombinant), Fc fusion protein | Hemophilia A | Factor XIII concentrate (human) | Congenital factor XII deficiency |
| Anti–inhibitor coagulant complex | Hemophilia A; B | Fish oil triglycerides | Parenteral nutrition–associated cholestasis |
| Asfotase alfa | Hypophosphatasia | Icatibant | Hereditary angioedema |
| Benznidazole | Chagas disease | Inotuzumab ozogamicin | Acute lymphoblastic leukemia |
| Blinatumomab | Acute lymphoblastic leukemia | Ivacaftor | Cystic fibrosis |
| Botulism antitoxin heptavalent (A, B, C, D, E, F, G) (equine) | Botulism | Lanadelumab-flyo | Hereditary angioedema |
| Burosumab-twza | X-linked hypophosphatemia | Lomitapide | Familial hypercholesterolemia |
| C1-esterase inhibitor (recombinant) | Hereditary angioedema | Lumacaftor–ivacaftor | Cystic fibrosis |
| C1-esterase-inhibitor (human, pasteurized) | Hereditary angioedema | Macimorelin acetate | Growth hormone deficiency |
| Cannabidiol | Lennox-Gastaut syndrome | Metreleptin | Lipodystrophy |
| Carglumic acid | N-acetylglutamate synthase deficiency | Migalastat hydrochloride | Fabry disease |
| Cerliponase alfa | Tripeptidyl peptidase 1 deficiency | Miltefosine | Leishmania donovani/braziliensis/guyanensis/panamensis |
| Cholic acid | Bile acid synthesis disorders | Mipomersen | Familial hypercholesterolemia |
| Clobazam | Lennox-Gastaut syndrome | Moxidectin | Onchocerca volvulus |
| Coagulation factor IX (recombinant) | Hemophilia B | Nusinersen | Spinal muscular atrophy |
| Coagulation factor IX (recombinant), Fc fusion protein | Hemophilia B | Pegvaliase-pqpz | Phenylketonuria |
| Coagulation factor X (human) | Factor X deficiency | Ponatinib | Acute lymphoblastic leukemia |
| Coagulation factor XIII A-subunit (recombinant) | Hemophilia A | Recombinant fusion protein; coagulation factor IX with albumin | Hemophilia B |
| Deferasirox | Non–transfusion-dependent thalassemia syndromes | Recombinant human acid α-glucosidase; alglucosidase alfa | Pompe disease |
| Deferiprone | Thalassemia syndromes | Recombinant von Willebrand factor | von Willebrand disease |
| Deflazacort | Duchenne muscular dystrophy | Sebelipase alfa | Lysosomal acid lipase |
| Dinutuximab | Neuroblastoma | Stiripentol | Dravet syndrome |
| Elapegademase-lvlr | Adenosine deaminase; severe combined immunodeficiency | Taliglucerase alfa | Type 1 Gaucher disease |
| Eliglustat | Gaucher disease | Tezacaftor and ivacaftor combination therapy | Cystic fibrosis |
| Elosulfase alfa | Mucopolysaccharidosis type IVA | Tisagenlecleucel | Acute lymphoblastic leukemia |
| Emapalumab-lzsg | Hemophagocytic lymphohistiocytosis | Uridine triacetate | Orotic aciduria |
| Emicizumab-kxwh | Hemophilia A | Varicella zoster immune globulin (human) | Varicella |
| Erwinia L-asparaginase | Acute lymphoblastic leukemia | Velaglucerase alfa | Gaucher disease |
| Eteplirsen | Duchenne muscular dystrophy | Vestronidase alfa-vjbk | Mucopolysaccharidosis type VII |
| Evolocumab | Familial hypercholesterolemia | Voretigene neparvovec-rzyl | Retinal dystrophy |