This list of orphan drugs is provided
for spelling reference only. Please read the notice at the bottom of this page
before using this list.
Generic Name: Daclizumab
Trade Name: Zenapax
Indication: Prevention of acute renal allograft rejection.
Generic Name: Daunorubicin citrate liposome injection
Trade Name: DaunoXome
Indication: Treatment of patients with advanced HIV-associated Kaposi's sarcoma.
Generic Name: Denileukin diftitox
Trade Name: Ontak
Indication: Treatment of patients with persistent or recurrent cutaneous T-cell lymphoma whose malignant cells express the CD25 component of the IL-2 receptor.
Generic Name: Desmopressin acetate
Trade Name:
Indication: Treatment of mild hemophilia A and von Willebrand's disease.
Generic Name: Dexrazoxane
Trade Name: Zinecard
Indication: For the prevention of cardiomyopathy associated with doxorubicin administration.
Generic Name: Diazepam viscous solution for rectal administration
Trade Name:
Indication: For the management of selected, refractory, patients with epilepsy, on stable regimens of antiepileptic drugs (AEDs), who require intermittent use of diazepam to control bouts of increased seizure activity.
Generic Name: Digoxin immune FAB (Ovine)
Trade Name: Digibind
Indication: Treatment of potentially life threatening digitalis intoxication in patients who are refractory to management by conventional therapy.
Generic Name: Dornase alfa
Trade Name: Pulmozyme
Indication: To reduce mucous viscosity and enable the clearance of airway secretions in patients with cystic fibrosis.
Generic Name: Doxorubicin liposome
Trade Name: Doxil
Indication: Treatment of ovarian cancer.
Generic Name: Dronabinol
Trade Name: Marinol
Indication: For the stimulation of appetite and prevention of weight loss in patients with a confirmed diagnosis of AIDS.
Generic Name: Eflornithine HCl
Trade Name: Ornidyl
Indication: Treatment of Trypanosoma brucei gambiense infection (sleeping sickness).
Generic Name: Epirubicin
Trade Name: Ellence
Indication: Treatment of breast cancer.
Generic Name: Epoetin alfa
Trade Name: Epogen
Indication: Treatment of anemia associated with end stage renal disease.
Generic Name: Epoetin alfa
Trade Name: Epogen
Indication: Treatment of anemia associated with HIV infection or HIV treatment.
Generic Name: Epoprostenol
Trade Name: Flolan
Indication: Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease.
Generic Name: Epoprostenol
Trade Name: Flolan
Indication: Treatment of primary pulmonary hypertension.
Generic Name: Etanercept
Trade Name: Enbrel
Indication: Reduction in signs and symptoms of moderately to severely active polyarticular-course juvenile rheumatoid arthritis in patients who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs.
Generic Name: Ethanolamine oleate
Trade Name: Ethamolin
Indication: Treatment of patients with esophageal varices that have recently bled, to prevent rebleeding.
Generic Name: Etidronate disodium
Trade Name: Didronel
Indication: Treatment of hypercalcemia of malignancy inadequately managed by dietary modification and/or oral hydration.
Generic Name: Exemestane
Trade Name: Aromasin
Indication: Treatment of advanced breast cancer in postmenopausal women whose disease has progressed following tamoxifen therapy.
Generic Name: Felbamate
Trade Name: Felbatol
Indication: Treatment of Lennox-Gastaut syndrome.
Generic Name: Filgrastim
Trade Name: Neupogen
Indication: Treatment of neutropenia associated with bone marrow transplants.
Generic Name: Filgrastim
Trade Name: Neupogen
Indication: Treatment of patients with severe chronic neutropenia (absolute neutrophil count less than 500/mm3).
Generic Name: Filgrastim
Trade Name: Neupogen
Indication: For use in the mobilization of peripheral blood progenitor cells for collection in patients who will receive myeloablative or myelosuppressive chemotherapy.
Generic Name: Filgrastim
Trade Name: Neupogen
Indication: Reduction in the duration of neutropenia, fever, antibiotic use, and hospitalization, following induction and consolidation treatment for acute myeloid leukemia.
Generic Name: Fludarabine phosphate
Trade Name: Fludara
Indication: Treatment of chronic lymphocytic leukemia (CLL), including refractory CLL.
Generic Name: Follitropin alfa, recombinant
Trade Name: Gonal-F
Indication: For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism.
Generic Name: Fomepizole
Trade Name: Antizole
Indication: Treatment of methanol or ethylene glycol poisoning.
Generic Name: Fosphenytoin
Trade Name: Cerebyx
Indication: For the acute treatment of patients with status epilepticus of the grand mal type.
Generic Name: Ganciclovir intravitreal implant
Trade Name: Vitrasert Implant
Indication: Treatment of cytomegalovirus retinitis.
Generic Name: Gemtuzumab zogamicin
Trade Name: Mylotarg
Indication: Treatment of CD33-positive acute myeloid leukemia.
Generic Name: Glatiramer acetate
Trade Name: Copaxone
Indication: Treatment of multiple sclerosis.
Generic Name: Gonadorelin acetate
Trade Name: Lutrepulse
Indication: For induction of ovulation in women with hypothalamic amenorrhea due to a deficiency or absence in the quantity or pulse pattern of endogenous GnRH secretion.
Generic Name: Halofantrine
Trade Name: Halfan
Indication: Treatment of mild to moderate acute malaria caused by susceptible strains of P. falciparum and P. vivax.
Generic Name: Hemin
Trade Name: Panhematin
Indication: Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.
Generic Name: Histrelin acetate
Trade Name: Supprelin Injection
Indication: Treatment of central precocious puberty.
Generic Name: Hydroxyurea
Trade Name: Droxia
Indication: Treatment of patients with sickle cell anemia as shown by the presence of hemoglobin S.
Generic Name: Idarubicin HCl for injection
Trade Name: Idamycin
Indication: Treatment of acute myelogenous leukemia, also referred to as acute nonlymphocytic leukemia.
Generic Name: Ifosfamide
Trade Name: Ifex
Indication: Treatment of testicular cancer.
Generic Name: Imiglucerase
Trade Name: Cerezyme
Indication: Replacement therapy in patients with types I, II, and III Gaucher's disease.
Generic Name: Immune globulin intravenous, human
Trade Name: Gamimune N
Indication: Infection prophylaxis in pediatric patients affected with the human immunodeficiency virus.
Generic Name: Infliximab
Trade Name: Remicade
Indication: Treatment of moderately to severely active Crohn's disease for the reduction of the signs and symptoms, in patients who have an inadequate response to conventional therapy; and treatment of patients with fistulizing Crohn's disease for the reduction in the number of draining enterocutaneous fistula(s).
Generic Name: Interferon alfa-2a
Trade Name: Roferon A
Indication: Treatment of chronic myelogenous leukemia.
Generic Name: Interferon alfa-2a (recombinant)
Trade Name: Roferon-A
Indication: Treatment of AIDS related Kaposi's sarcoma.
Generic Name: Interferon alfa-2b (recombinant)
Trade Name: Intron A
Indication: Treatment of AIDS-related Kaposi's sarcoma.
Generic Name: Interferon beta-1a
Trade Name:
Indication: Treatment of multiple sclerosis.
Generic Name: Interferon beta-1b
Trade Name: Betaseron
Indication: Treatment of multiple sclerosis.
Generic Name: Interferon gamma 1-b
Trade Name: Actimmune
Indication: Treatment of chronic granulomatous disease.
Generic Name: Interferon gamma-1b
Trade Name: Actimmune
Indication: Delaying time to disease progression in patients with severe, malignant osteopetrosis.
Generic Name: Iobenguane Sulfate I 131
Trade Name:
Indication: For use as a diagnostic adjunct in patients with pheochromocytoma.
Please Click HERE for
a comma-delimited list of the orphan drugs that you may add to your speller.
List made available by express
written permission of
NORD — National Organization for Rare Disorders, Inc.
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