The Food and Drug Administration has charged the Office of Orphan Products Development (OOPD) to dedicate its mission to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD interacts with the medical and research communities, professional organizations, academia, governmental agencies, and the pharmaceutical industry, as well as rare disease groups.

 

The OOPD administers the major provisions of the Orphan Drug Act (ODA) which provide incentives for sponsors to develop products for rare diseases. The ODA has been very successful - more than 200 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.

There is one or more orphan products that have been FDA approved for the treatment of the diseases listed below.

A  |

B  |

C  |

D  |

E  |

F  |

G  |

H  |

I  |

J  |

K  |

L  |

M  |

N  |

O  |

P  |

Q  |

R  |

S  |

T  |

U  |

V  |

W  |

X  |

Y  |

Z  |

0-49

A
Acral lentiginous melanoma
Acromegaly
Acute intermittent porphyria
Acute lymphoblastic leukemia
Acute lymphoblastic leukemia, childhood
Acute myeloid leukemia, adult
Acute promyelocytic leukemia
Adenosine deaminase deficiency
Alpha 1-antitrypsin deficiency
Alveolar capillary dysplasia
Amebiasis
Amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis type 10
Amyotrophic lateral sclerosis type 11
Amyotrophic lateral sclerosis type 2
Amyotrophic lateral sclerosis type 3
Amyotrophic lateral sclerosis type 4
Amyotrophic lateral sclerosis type 5
Amyotrophic lateral sclerosis type 6
Amyotrophic lateral sclerosis type 7
Amyotrophic lateral sclerosis type 8
Amyotrophic lateral sclerosis type 9
Anaplastic large cell lymphoma
Anterior uveitis
Anthrax
Aortic aneurysm, familial thoracic 4
Arginase deficiency
Argininosuccinic aciduria
Atypical hemolytic uremic syndrome
Autosomal recessive juvenile Parkinson disease

B (Back to Top)
Barrett syndrome
B-cell lymphomas
Bidirectional tachycardia
Blepharospasm
Botulism

C (Back to Top)
Carbamoyl phosphate synthetase 1 deficiency
Carcinoid syndrome
Carcinoid tumor
Cardiomyopathy due to anthracyclines
Catecholaminergic polymorphic ventricular tachycardia
CDK4 linked melanoma
Cerebellar ataxia and hypogonadotropic hypogonadism
Chang Davidson Carlson syndrome
Char syndrome
Chromophil renal cell carcinoma
Chromophobe renal cell carcinoma
Chronic granulomatous disease
Chronic Infantile Neurological Cutaneous Articular syndrome
Chronic inflammatory demyelinating polyneuropathy
Chronic lymphocytic leukemia
Chronic myeloid leukemia
Chronic myeloproliferative disorders
Chudley Rozdilsky syndrome
Citrullinemia type I
Classic Kaposi sarcoma
Clear cell renal cell carcinoma
Coccidioidomycosis
Congenital antithrombin deficiency
Congenital antithrombin deficiency type 2
Congenital antithrombin deficiency type 3
Congenital herpes simplex
Congenital sucrase-isomaltase deficiency
Crohn's disease
Cryptococcosis
Cryptosporidiosis
Cushing's syndrome
Cutaneous T-cell lymphoma
Cystic fibrosis
Cysticercosis
Cystinosis
Cystinuria
Cytomegalovirus retinitis

D (Back to Top)
Dermatofibrosarcoma protuberans
Disseminated infection with mycobacterium avium complex
Dystonia 1
Dystonia 2, torsion, autosomal recessive
Dystonia 7, torsion

E (Back to Top)
Endemic Kaposi sarcoma
Epileptic encephalopathy Lennox-Gastaut type
Esophageal cancer
Esophageal varices
Essential thrombocythemia
Ewing's family of tumors
Ewing's sarcoma

F (Back to Top)
Fabry disease
Factor VII deficiency
Factor XIII deficiency
Familial cold autoinflammatory syndrome
Familial colorectal cancer
Familial isolated hyperparathyroidism
Familial Mediterranean fever
Familial myelofibrosis
Familial prostate cancer
Familial renal cell carcinoma
Familial ventricular tachycardia
Fibrinogen deficiency, congenital
Focal dystonia

G (Back to Top)
Gastrointestinal Stromal Tumors
Gaucher disease
Gaucher disease type 1
Glioblastoma
Glioma
Glycogen storage disease type 2
Gorlin Chaudhry Moss syndrome
Growth hormone deficiency
Growth hormone insensitivity with immunodeficiency

H (Back to Top)
Hairy cell leukemia
Hansen's disease
Hemolytic uremic syndrome, atypical, childhood
Hemophilia A, congenital
Hemophilia B
Heparin-induced thrombocytopenia
Hepatic encephalopathy
Hepatocellular carcinoma (fibrolamellar variant)
Hereditary angioedema
Herpes simiae (B virus)
Herpes simplex encephalitis
Herpes zoster oticus
Herpesvirus simiae B virus
Herpetic keratitis
Hodgkin lymphoma
Homocysteinemia due to MTHFR deficiency
Homocystinuria due to CBS deficiency
Homocystinuria due to defect in methylation cbl e
Homocystinuria due to defect in methylation cbl g
Huntington disease
Hurthle cell thyroid cancer
Hydatidosis
Hypereosinophilic syndrome
Hyperparathyroidism, neonatal severe primary
Hyperparathyroidism, primary
Hypogonadotropic hypogonadism without anosmia, X-linked
Hypothalamic dysfunction

I (Back to Top)
Idiopathic pulmonary hypertension
Idiopathic thrombocytopenic purpura
Infantile apnea
Interstitial cystitis
Intraocular melanoma

J (Back to Top)
Juvenile idiopathic arthritis

K (Back to Top)
Kallmann syndrome 2
Kallmann syndrome 3
Kidney cancer

L (Back to Top)
Laron syndrome
Leiomyomatosis and renal cell cancer, hereditary
Leishmaniasis
Lentigo maligna melanoma
Leukemia, B-cell, chronic
Liver cancer
Lymphoblastic lymphoma
Lynch syndrome

M (Back to Top)
Malaria
Malignant melanoma, childhood
Malignant mesothelioma
Mastocytosis
Mastocytosis cutaneous with short stature conductive hearing loss and microtia
Melanoma astrocytoma syndrome
Melanoma, familial
Methylmalonic acidemia with homocystinuria
Methylmalonic aciduria with homocystinuria cbl f
Methylmalonicacidemia with homocystinuria cbl d
Microscopic polyangiitis
Muckle-Wells syndrome
Mucopolysaccharidosis type I
Mucopolysaccharidosis type II
Mucopolysaccharidosis type VI
Multiple endocrine neoplasia type 2A
Multiple myeloma
Multiple sclerosis
Multiple system atrophy (MSA) with orthostatic hypotension
Myelodysplastic syndromes
Myelodysplastic/myeloproliferative disease
Myelofibrosis
Myopathic carnitine deficiency

N (Back to Top)
Narcolepsy
Natal teeth, intestinal pseudoobstruction and patent ductus
Nephropathic cystinosis
Neuroblastoma
Nodular melanoma
Non-small cell lung cancer, childhood
Noonan syndrome 1

O (Back to Top)
Ocular melanoma
Ornithine transcarbamylase deficiency
Oslam syndrome
Osteopetrosis
Osteopetrosis and infantile neuroaxonal dystrophy
Osteopetrosis autosomal dominant type 1
Osteopetrosis autosomal dominant type 2
Osteopetrosis autosomal recessive 3
Osteopetrosis autosomal recessive 5
Osteopetrosis autosomal recessive 7
Osteosarcoma
Ovarian cancer

P (Back to Top)
Paget disease of bone
Paget disease of bone, familial
Panuveitis
Papillary renal cell carcinoma
Parathyroid carcinoma
Parkinson disease
Parkinson disease type 3
Parkinson disease type 9
Paroxysmal nocturnal hemoglobinuria
Patent ductus arteriosus
Pediatric Crohns disease
Pediatric ulcerative colitis
Peripheral T-cell lymphoma
Phenylketonuria
Pheochromocytoma
Pheochromocytoma, childhood
Pheochromocytoma-islet cell tumor syndrome
Pneumocystic carinii pneumonia
Prader-Willi syndrome
Precocious puberty
Premature aging Okamoto type
Primary biliary cirrhosis
Primary carnitine deficiency
Primary gastrointestinal melanoma
Primary malignant melanoma of the cervix
Primary malignant melanoma of the conjunctiva
Protein C deficiency

R (Back to Top)
Renal cell carcinoma 4
Respiratory distress syndrome, infant
ROHHAD

S (Back to Top)
Severe combined immunodeficiency
Short bowel syndrome
Sickle cell anemia
Sjogren syndrome
Slti Salem syndrome
Soft tissue sarcoma
Soft tissue sarcoma childhood
Spinal cord neoplasm
Squamous cell carcinoma
Squamous cell carcinoma of the head and neck
Status epilepticus
Superficial spreading melanoma

T (Back to Top)
T-cell lymphoma 1A
Testicular cancer
Thyroid cancer, anaplastic
Thyroid cancer, follicular
Thyroid cancer, medullary
Tièche-Jadassohn nevus
Torsion dystonia
Transverse myelitis
Trypanosomiasis, Human West-African
Tuberculosis
Tuberous sclerosis
Tuberous sclerosis, type 1
Tuberous sclerosis, type 2
Turner syndrome
Tyrosinemia type 1

U (Back to Top)
Urea cycle disorders
Uterine sarcoma

V (Back to Top)
Ventricular fibrillation, idiopathic
Vernal keratoconjunctivitis
VIPoma
Von Willebrand disease

W (Back to Top)
Wegener's granulomatosis
Wilson disease

Z (Back to Top)
Zollinger-Ellison syndrome

Go directly to the Orphan Drug Product Designation database to search by specific dates, products, and/or indications. Search results from the Orphan Drug Product Designation database can be provided as a condensed list, detailed list, or an Excel spreadsheet.