Medical Products List
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
Aceruloplasminemia
Acral lentiginous melanoma
Acromegaly
Acute intermittent porphyria
Acute lymphoblastic leukemia
Acute lymphoblastic leukemia, childhood
Acute myeloid leukemia, adult
Acute promyelocytic leukemia
Adenosine deaminase deficiency
Aggressive systemic mastocytosis
Albers-Schonberg disease
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
Anti-factor 8 autoimmunization
Aortic aneurysm, familial thoracic 4
Atypical mole syndrome
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Barrett syndrome
B-cell lymphomas
Benign essential blepharospasm
Bidirectional tachycardia
Botulism
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Carcinoid syndrome
Carcinoid tumor
Carcinoma, squamous cell
Cardiomyopathy due to anthracyclines
CDK4 linked melanoma
Chang Davidson Carlson syndrome
Char syndrome
Chromophil renal cell carcinoma
Chromophobe renal cell carcinoma
Chronic granulomatous disease
Chronic inflammatory demyelinating polyneuropathy
Chronic lymphocytic leukemia
Chronic myeloid leukemia
Chronic neutropenia
Chronic, Infantile, Neurological, Cutaneous, Articular syndrome
Chudley Rozdilsky syndrome
Classic Kaposi sarcoma
Clear cell renal cell carcinoma
Colorectal cancer, hereditary nonpolyposis, type 1
Colorectal cancer, hereditary nonpolyposis, type 2
Congenital antithrombin deficiency
Congenital antithrombin deficiency type 2
Congenital antithrombin deficiency type 3
Congenital sucrose isomaltose malabsorption
Congenital thrombotic disease, due to Protein C deficiency
Crohn's disease
Cryptococcosis
Cryptosporidiosis
Cutaneous T-cell lymphoma
Cystic fibrosis
Cysticercosis
Cystinuria
Cytomegalovirus retinitis
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Dermatofibrosarcoma protuberans
Disseminated infection with mycobacterium avium complex
Dystonia 1
Dystonia 2, torsion, autosomal recessive
Dystonia 7, torsion
Dystonia, focal, task-specific
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Epileptic encephalopathy, Lennox-Gastaut type
Esophageal cancer
Esophageal varices
Essential thrombocythemia
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Fabry disease
Factor VII deficiency
Familial cold autoinflammatory syndrome
Familial colorectal cancer
Familial Mediterranean fever
Familial Paget disease of bone
Familial prostate cancer
Familial renal cell carcinoma
Familial ventricular tachycardia
Fibrinogen deficiency, congenital
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Gastrointestinal Stromal Tumors
Gaucher Disease
Gaucher disease type 1
Gelineau syndrome
Giardiasis
Glioma
Glycogen storage disease type 2
Gorlin Chaudhry Moss syndrome
Growth hormone deficiency
Growth hormone insensitivity with immunodeficiency
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Hairy cell leukemia
Hansen's disease
Hemophilia A, congenital
Hemophilia B
Heparin induced thrombocytopenia
Hepatocellular carcinoma (fibrolamellar variant)
Hepatocellular carcinoma, adult
Hereditary angioedema
Hereditary carnitine deficiency
Herpetic keratitis
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
Hydatidosis
Hypogonadotropic hypogonadism without anosmia, X-linked
Hypogonadotropic hypogonadism-anosmia
Hypogonadotropic hypogonadism-anosmia, X-linked
Hypothalamic dysfunction
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Idiopathic hypereosinophilic syndrome
Idiopathic pulmonary hypertension
Infantile apnea
Infantile spasms
Interstitial cystitis
Intraocular melanoma
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Juvenile rheumatoid arthritis
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Kallmann syndrome 2
Kallmann syndrome 3
Kidney cancer
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Laron syndrome
Leiomyomatosis and renal cell cancer, hereditary
Leishmaniasis
Lentigo maligna melanoma
Leukemia, B-cell, chronic
Luteinizing hormone releasing hormone, deficiency of with ataxia
Lymphoblastic lymphoma
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Malaria
Malignant melanoma, childhood
Malignant paroxysmal ventricular tachycardia
Mast cell disease
Mastocytosis, cutaneous, with short stature, conductive hearing loss and microtia
Melanoma astrocytoma syndrome
Melanoma of the choroid
Melanoma of the ciliary body
Melanoma of the iris
Melanoma, familial
Mesothelioma, adult malignant
Methylmalonic acidemia with homocystinuria
Methylmalonicacidemia with homocystinuria cbl d
Methylmalonicaciduria with homocystinuria cbl f
Muckle-Wells syndrome
Mucopolysaccharidosis type I
Mucopolysaccharidosis type II
Mucopolysaccharidosis type VI
Multiple endocrine neoplasia type 2A
Multiple myeloma
Multiple sclerosis
Myelodysplastic syndromes
Myopathic carnitine deficiency
Myxedema
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Natal teeth, intestinal pseudoobstruction and patent ductus
Nephropathic cystinosis
Neuroblastoma
Neutropenia, severe chronic
Nodular melanoma
Noonan syndrome
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Ocular melanoma
Oslam syndrome
Osteopetrosis
Osteopetrosis and infantile neuroaxonal dystrophy
Osteopetrosis autosomal dominant type 1
Osteopetrosis lethal
Osteopetrosis with renal tubular acidosis
Osteopetrosis, renal tubular acidosis and basal ganglia calcification
Osteosarcoma
Ovarian cancer
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Paget disease
Pantothenate kinase-associated neurodegeneration
Papillary renal cell carcinoma
Parathyroid carcinoma
Parkinson disease
Parkinson disease 3
Parkinson disease 9
Parkinson disease, juvenile, autosomal recessive
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
Polymorphic catecholergic ventricular tachycardia
Prader-Willi syndrome
Precocious puberty
Premature aging, Okamoto type
Primary biliary cirrhosis
Primary gastrointestinal melanoma
Primary malignant melanoma of the conjunctiva
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Renal cell carcinoma 4
Respiratory distress syndrome, infant
ROHHAD
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Serious digitalis intoxication
Severe combined immunodeficiency
Short bowel syndrome
Shy-Drager syndrome
Sickle cell anemia
Sjogren syndrome
Slti Salem syndrome
Spinal cord neoplasm
Squamous cell carcinoma of the head and neck
Status epilepticus
Superficial spreading melanoma
Systemic carnitine deficiency
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T-cell lymphoma 1A
Testicular cancer
Thrombocytopenic purpura, autoimmune
Thyroid cancer, anaplastic
Thyroid cancer, familial medullary
Thyroid cancer, follicular
Thyroid cancer, Hurthle cell
Thyroid cancer, medullary
Thyroid cancer, papillary
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
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Urea cycle disorders
Uveal melanoma
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Ventricular fibrillation, idiopathic
Vernal keratoconjunctivitis
VIPoma
Von Willebrand disease
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Wilson disease
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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.