http://www.raredr.com/resource-guide/2016/appendix-a:-fda-approved-orphan-drugs
Appendix A: FDA Approved Orphan Drugs


 
A
 
Acquired hypoprothrombinemia                                        
Prothrombin complex                                                              
                                                                                                                                                                         

Acromegaly                                                                              
Octeotide
Pasireotide
Pegvisomant

Acute intermittent porphyria                                                 
Hemin injection
 
Acute lymphoblastic leukemia                                            
Asparaginae E chrys.                                                              
Clorarabine                                                                                
Dasatinib
Imatinib mesylate
Nelarabine
Pegaspargase 
Ponatinib
Vincristine sulfate 
 
Acute lymphoblastic leukemia, childhood                       
Mercaptopurine                                                                         
Teniposide 
 
Acute lymphocytic leukemia
Blinatumomab
Cholic acid
 
Acute myeloid leukemia, adult                                             
Filgastim 
Idarubicin 
Mitoxantrone 
Sargramostim
 
Acute promyelocytic leukemia                                            
Arsenic trioxide 
Tretinoin                                                                                     
 

Adenosine deaminase deficiency                                      
Pegademase bovine 
 

Adult-onset citrullinemia type II                                           
Glycerol phenylbutyrate 
 

Alpha 1-antitrypsin deficiency                                             
Alpha1 proteinase inhibitor 
 

Alveolar capillary dysplasia                                                 
Nitric oxide 
 

Amebiasis                                                                                 
Tinidazole 
 

Amyotrophic lateral sclerosis (types 2-11) 
Riluzole 
 

Anaplastic large cell lymphoma                                          
Brentuximab
 
Anterior uveitis                                                                        
Difluprednate 
 

Anthrax                                                                                      
Raxibacumab 
Anthrax immune globulin
 
Aortic aneurysm, familial thoracic 4                                   
Ibufrofen lysine
 
Aspergillosis, invasive
Isavuconazonium sulfate

Atypical hemolytic uremic syndrome                                
Eculizumab 
 

Autosomal recessive juvenile Parkinson disease         
Apomorphine HCl 
 
B
 

Barrett syndrome
Porfimer 
 

B-cell lymphomas                                                                   
Ibritumoab tiuxetan
Rituximab 
Tositumomab
                                                                                                     
Bidirectional tachycardia                                                      
Amiodarone
Solalol
 
Blepharospasm                                                                       
Onabotulinumtoxin A 
                                                                                                     

Botulism                                                                                    
Botulism antitoxin 
 
C
 

Carcinoid syndrome                                                              
Octeotide
 
Carcinoid tumor                                                                      
Octeotide
 
Cardiomyopathy due to anthracyclines                           
Dexrazoxane
 
Castleman’s Disease                                                             
Siltuximab
 
Catecholaminergic polymorphic ventricular tachycardia
Amiodarone 
Sotalol
 
CDK4 linked melanoma                                                         
Aldesleukin                                                                                
 

Char syndrome                                                                        
Ibuprofen lysine 
 

Chromophil renal cell carcinoma                                        
Aldesleukin
Sorafenib 
Temsirolimus 
 

Chronic granulomatous disease                                        
Interferon gamma-1b
 
Chronic inflammatory demyelinating polyneuropathy
 Immune globulin 
 

Chronic lymphocytic leukemia                                            
Alemtuzumab
Bendamustine
Fludarabine 
Fludarabine (oral) 
Ibrutinib
Idelalisib
Imbruvica
Obinutuzumab
Ofatumumab 
                                                                                                     
Chronic myeloid leukemia                                                    
Bosutinib
Dasatinib
Imatinib 
Interferon alfa-2a 
Nilotinib 
Omacetaxine  
Ponatinib 
 

Chronic myeloproliferative disorders                                
Imatinib                                                                                       
 

Classic Kaposi sarcoma                                                       
Alitretinoin
Daunorubicin
Interferon alfa-2a
Interferon alfa-2b                                                                      
Paclitaxel
Pazopanil 
 

Clear cell renal cell carcinoma                                             
Aldesleukin 
Sorafenib
Temsirolismus 
 

Congenital antithrombin deficiency                                   
Antithrombin 
 

Congenital antithrombin deficiency type 2                      
Antithrombin
 
Congenital antithrombin deficiency type 3                      
Antithrombin
Anitthrombin III
                                                                                                     
Congenital generalized lipodystrophy type 1                 
Metreleptin 
 
Congenital generalized lipodystrophy type 2                 
Metreleptin 
 
Congenital generalized lipodystrophy type 4
Metreleptin 
 
Congenital sucrase-isomaltase deficiency                      
Sacrosidase
 
Crohn's disease                                                                      
Adalimumab
Infliximab
 
Cryopyrin-associated periodic syndrome                       
Canakinumab
 
Cryptococcosis                                                                       
Amphotericin B  
 

Cryptosporidiosis                                                                   
Nitazoxanide 
 

Cushing's syndrome                                                             
Mifepristone 
Pasireotide
 
Cutaneous T-cell lymphoma                                                
Bexarotene 
Denileukin diftitox
Romidepsin 
 

Cystic fibrosis                                                                          
Aztreonam 
Dornase alfa 
Ivacaftor 
Tobramycin 
 

Cysticercosis                                                                           
Albenadazole 
 

Cystinosis                                                                                 
Cysteamine 
 

Cystinuria                                                                                  
Tiopronin 
 

Cytomegalovirus retinitis                                                      
Gaciclovir 
 
D
 

Dermatofibrosarcoma protuberans                                   
Imatinib 
 

Diffuse gastric cancer                                                            
Ramacirumab
 
Disseminated infection with mycobacterium avium complex                                          
Rifabutin 
 

Dopamine B hydroxylase deficiency                                 
Droxidopa
 
Dystonia 1                                                                                 
Botulinum toxin A 
 

Dystonia 2, torsion, autosomal recessive                        
Botulinum toxin A 
 

Dystonia 7, torsion                                                                 
Botulinum toxin A
 
E
 

Endemic Kaposi sarcoma                                                    
Pazopanib
                                                                                                     
Esophageal cancer                                                                
Porfimer
 
Esophageal varices                                                                
Ethanolamine oleate
 
Essential thrombocythemia                                                 
Anagrelide 
 

Ewing's family of tumors                                                      
Pazopanib
Denosumab
 
Ewing's sarcoma                                                                    
Pazopanib
 
F
 

Fabry disease                                                                          
Ceramide trihexosidase
 
Factor VII deficiency                                                               
Coagulation factor V11a 
 

Factor XIII deficiency                                                              
Factor XIII
Factor XIII A subunit
 
Fallopian tube carcinoma
Bevacizumab

Familial colorectal cancer                                                     
Leucovorin  
 

Familial isolated hyperparathyroidism                              
Cinacalcet
 
Familial Mediterranean fever                                                
Colchicine 
 
Familial myelofibrosis                                                            
Ruxolitinib 
 

Familial prostate cancer                                                        
Miloxantrone 
 

Familial renal cell carcinoma                                                
Aldeleukin 
Sorafenib 
Temsirolimus 
 

Familial ventricular tachycardia                                          
Amiodarone
Sotalol HCl
Sotalol
 
Fibrinogen deficiency, congenital                                      
Fibrinogen  
 

Fibrolamellar hepatocellular carcinoma                           
Sorafenib
 
Focal dystonia                                                                         
Botulinum toxin type A
Botulinum toxin type B 
 

Follicular B-cell non-Hodgkin lymphoma
Idelalisib
 
G
 

Gastric cancer (advanced)                                                   
Ramucirumab
 
Gastrointestinal stromal tumors                                         
Ianreotide
Imatinib
Regorafenib 
                                                                                                     

Gaucher disease                                                                     
Eliglustat
Imiglucerase
Miglusatat 
Taliglucerase alfa
Velaglucerase
 
Glioblastoma                                                                            
Polifeprosan 20
 
Glioma                                                                                        
Polifeprosan 20                                                                                                     
Temozolomide 
 

Glycogen storage disease type 2 (Pompe)                      
Alphaglucosidase 
 

Gorlin Chaudhry Moss syndrome                                      
Ibbuprofen lysine 
 

Granulomatosis with polyangiitis (Wegener’s)               
Rituximab 
 

Growth hormone deficiency                                                
Sermorelin
Somatrem
Somatropin
Genotropin 
Humatrope 
Norditropin
Nutropin 
Saizen 
 

Growth hormone insensitivity with immunodeficiency
Mecasermin
 
H
 

Hairy cell leukemia                                                                  
Leustatin 
Pentostatin 
 

Hansen's disease                                                                   
Clofazimine                                                                               
Thalomide 
 

Hemolytic uremic syndrome, atypical, childhood          
Eculizumab 
 

Hemophilia A, congenital                                                      
Eloctate
Kogenate                                                                                    
Refacto 
Porcine sequence
Anti-inhibitor coagulant
Coagulation Factor VIIa
Desmopressin 
 

Hemophilia B                                                                            
Coagulation Factor IX
  Alphanine
  Alprolix
  BeneFix 
  Mononine
  Rixubis
Coagulation Factor VIIa 
Factor VIIIa Inhibitor
 
Heparin-induced thrombocytopenia                                 
Lepirudin 
 
Hepatocellular carcinoma                                                     
Sorafenib
 
Hereditary angioedema                                                         
C1 esterase inh (human) 
C1 esterase inh (recomb)
Ecallantide 
Icatibant 
                                                                                                     

Hereditary leiomyomatosis and renal cell cancer          
Aldesleukin
Sorafenib
Temsirolimus                                                                             
 

Hodgkin lymphoma                                                                
Brentuximab 
 

Homocysteinemia                                                                   
Betaine 
 
Huntington disease                                                                
Tetrabenazine 
 

Hurthle cell thyroid cancer                                                   
Thyrotropin alfa
 
Hydatidosis                                                                              
Albendazole 
 

Hypereosinophilic syndrome                                              
Imatinib 
Cinacalcet 
 

Hypogonadotropic hypogonadism
Luteinizing hormone
 
Hypoparathyroidism
Parathyroid hormone

Hypothalamic dysfunction                                                   
Follitropin alfa 
Gonadorelin
 
I
 

Idiopathic pulmonary fibrosis
Nintedanib
Pirfenidone
 
Idiopathic thrombocytopenic purpura                              
Eltrombopag 
Human immunoglobulin
Rho (D) immunoglobulin                                                        
Romiplostim 
 
Infantile apnea                                                                         
Caffiene 
 

Infantile hemangiomas                                                          
Propranolol HCl
 
Interstitial cystitis                                                                    
Pentosan polysulfate 
 
J


Japanese encephalitis                                                           
Vaccine 
 

Juvenile idiopathic arthritis                                                  
Adalimumab                                                                              
Canakinumab
Etanercept
Meloxicam 
Tocilizumab
 
K
 

Kidney cancer                                                                          
Aldesleukin 
Sorafenib 
Temsirolimus
 
L
 

Laron syndrome                                                                      
Mecasermin 
                                                                                                     

Leishmaniasis                                                                          
Amphotericin B 
Miltefosine
 
Lennox-Gastaut syndrome                                                  
Rufinamide 
Felbamate 
Lamotrigine 
Topiramate
                                                                                                     
Leukemia, B-cell, chronic                                                     
Fludarabine
 
Liver cancer                                                                              
Sorafenib 
 

Lymphoblastic lymphoma                                                    
Nelarabine
 
 
M


Malaria                                                                                        
Halofantrine
Melfloquin 
Quinin sulfate
 
Malignant hyperthermia                                                      
Dantrolene
 
Malignant melanoma, childhood                                         
Aldesleukin 
Ipilimumab
Peginterferon alfa 2b 
 

Malignant mesothelioma                                                       
Pemetrexed 
 

Mantle cell lymphoma                                                            
Bortezomib
Imbrutinib
Lenalidomide
 
Mastocytosis                                                                            
Cromolyn 
 

Melanoma astrocytoma syndrome                                     
Aldesleukin 
 

Melanoma                                                                                  
Aldesleukin 
Dabrafenib
Ipilimumab
Nivolumab
Pegintron alfa 2b
Pembrolizumab
Technetium Tc99m
Trametiinib
                                                                                                     
Methylmalonic acidemia / aciduria                                     
Betaine anhydrous
 
Microscopic polyangiitis                                                       
Rituximab 
 
Muckle-Wells syndrome                                                        
Rilonacept 
 
Mucopolysaccharidosis type I                                            
Laronidase 
 

Mucopolysaccharidosis type II                                           
Idursulfase 
 

Mucopolysaccharidosis type VI                                          
Galsulfase 
 

Mucopolysaccharidosis type VIA                                       
Elosulfase alfa 
 

Mucormycosis, invasive
Isavuconazonium sulfate
 
Multiple myeloma                                                                    
Bortezomid 
Carfilzomib
Doxirubicin 
Lenalidomide 
Melphalan
Panobinostat
Pomalidomide 
Thalomidomide 
 

Mycosis fungoides                                                                 
Mechlorethamine 
 

Myelodysplastic syndromes                                                
Azacitidine 
Decitabine 
Lenalidomide 
 
Myelodysplastic/myeloproliferative disease                    
Imatinib 
 

Myelofibrosis                                                                            
Ruxolitinib
 
Myopathic carnitine deficiency                                           
Levocarnitine 
 

Myelosuppression
Filgrastim
 
N
 

N-acetylglutamate synthetase deficiency                        
Carglumic acid
 
Narcolepsy                                                                               
Modafinil 
Sodium oxybate
                                                                                                                                   
Nephropathic cystinosis                                                       
Cysteamine
                                                                                                     
Neurogenic orthostatic hypotension                               
Droxidopa
 
Nodular melanoma                                                                 
Aldesleukin 
 

Non-24 hour sleep-wake disorder                                      
Tasimelteon
 
Non-small cell lung cancer, childhood                              
Crizotinib 
 

Non-small cell lung cancer, rare form                                
Ceritinib
 
Noonan syndrome 1                                                              
Somatropin 
 
O
 

Ocular melanoma                                                                    
Aldesleukin 
 

Ornithine transcarbamylase deficiency                            
Glycerol phenylbutyrate 
 

Oslam syndrome                                                                     
Levoleucovorin 
Methotrexate 
 
Osteopetrosis                                                                          
Interferon gamma-1b 
Osteosarcoma                                                                           
Levoleucovorin
Methotrexate 
 
Ovarian cancer                                                                        
Altretamine 
Amifostine
Bevacizumab
Doxorubicin 
Olaparib
 
P
 

Paget disease of bone                                                           
Calcitonin 
 
Pancreatic cancer                                                                   
Paclitaxel
 
Panuveitis                                                                                 
Dexamethasone 
 

Papillary renal cell carcinoma                                              
Aldesleukin 
Sorafenib 
Temsirolimus
 
Parathyroid carcinoma                                                          
Cinacalcet 
 

Parkinson disease type 3 or 9                                             
Apomorphine
Levodopa and carbidopa
 
Paroxysmal nocturnal hemoglobinuria                            
Eculizumab 
 

Patent ductus arteriosus                                                      
Ibuprofen 
 

Pediatric Crohns disease                                                     
Infliximab 
 

Pediatric hypertension                                                          
Enalapril maleate
 
Pediatric neuroblastoma
Dinutuximab

Pediatric ulcerative colitis                                                     
Balsalazide 
Infliximab 
 

Peripheral T-cell lymphoma                                                 
Belinostat
Pralatrexate 
 
Peritoneal carcinoma
Bevacizumab

Peyronie’s disease                                                                 
Collagenase clostridium
 
Phenylketonuria                                                                      
Sapropterin 
 

Pneumocystic carinii pneumonia                                       
Atovaquone 
Pentamidine 
Trimetreaxate
 
Poison, rattlesnake
Polyvalent antivenom

Polycythemia vera
Ruxolitinib 
 
Pompe disease                                                                       
Alglucosidase alfa

Postherpetic neuralgia                                                          
Capsaicin
 
Prader-Willi syndrome                                                           
Somatropin
                                                                                                     
Precocious puberty                                                                
Histrelin 
Leuprolide
Nafarelin
 
Premature aging, Okamoto type                                          
Levoleucovorin
Methotrexate 
 
Primary biliary cirrhosis                                                        
Ursodiol
                                                                                                     
Primary carnitine deficiency                                                
Levocarnitine
 
Primary gastrointestinal melanoma                                   
Aldesleukin 
 

 Primary malignant melanoma of the cervix                       
Aldesleukin 
 

Primary malignant melanoma of the conjunctiva           
Aldesleukin 
 

Protein C deficiency                                                               
Protein C 
 

Pulmonary arterial hypertension                                        
Ambrisentan
Bosentan 
Epoprostenol 
Ilioprost 
Macitentan
Riociguat 
Treprostinil 
 
R
 

Renal cell carcinoma 4                                                          
Aldesleukin
Sorafenib
Temsirolimus
 
Renal replacement therapy
Phoxilium
 
Respiratory distress syndrome, infant                              
Calfactant 
Colfosceril palmitate
Poractant alfa 
 
 
S
 

Severe combined immunodeficiency                                
Pegademase bovine 
 

Short bowel syndrome                                                          
L-glutamine 
Somatropin
Teduglutide 
 

Sickle cell anemia                                                                   
Hydroxyurea 
 

Sjogren syndrome                                                                  
Pilocarpine 
 
Soft tissue sarcoma                                                               
Pazopanib
 
Spinal cord neoplasm                                                            
Baclofen
 
Squamous cell carcinoma                                                    
Cetuximab 
 

Status epilepticus                                                                   
Fosphenytoin 
 
 
T
 

T-cell lymphoma 1A                                                                
Pralatrexate
 
Testicular cancer                                                                    
Ifosfamide
 
Thyroid cancer, anaplastic    
Lenvatinib                                           
Thyrotropin alfa 
 
Thyroid cancer, follicular     
Lenvatinib                                                  
Thyrotropin alfa 
 
Thyroid cancer, medullary                                                   
Cabozantinib
Lenvatinib 
Thyrotropin alfa 
Vandetanib
 
Thyroid cancer, recurrent or metastatic 
Lenvatinib                        
Sorafenib
 
Tourette’s syndrome
Aripiprazole

Torsion dystonia                                                                     
Onabotulinem toxin A 
 

Transverse myelitis                                                                
Baclofen
 
Tuberculosis                                                                            
Aminosalicylic acid                                                                   
Bedaquiline 
Rifapentine 
Rifapentine, isoniazide, pyraz
                                                                                                     
Tuberous sclerosis                                                                
Everolimus
Vigabatrin 
 
Turner syndrome                                                                    
Somatropin                                                                                
 

Tyrosinemia type 1                                                                 
Nitisinone 
 
U
 

Urea cycle disorders                                                              
Glycerol phenylbutyrate
Sodium phenylacetate 
Sodium phenylbutyrate 
 
V
 

Ventricular fibrillation, idiopathic                                        
Sotalol
 
Vernal keratoconjunctivitis                                                  
Lodoxamide tromethamine 
 

Visceral leishmaniasis                                                           
Miltefosine
 
Von Willebrand disease                                                        
Antihemophilic Factor 
Desmopressin 
Coagulation Factor VIII 
 
W
 

Wilson disease                                                                        
Trientine 
Zinc acetate
 
Z
 

Zollinger-Ellison syndrome                                                  
Secretin



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