NORD Orphan Drug List

NORD Orphan Drugs:

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: Albendazole
Trade Name: Albenza
Indication: Treatment of hydatid disease (cystic echinococcosis due to E. granulosus larvae or alveolar echinococcosis due to E. multilocularis larvae).

Generic Name: Albendazole
Trade Name: Albenza
Indication: Treatment of neurocysticercosis due to Taenia solium as: 1) chemotherapy of parenchymal, subarachnoidal and racemose (cysts in spinal fluid) neurocysticercosis in symptomatic cases and 2) prophylaxis of epilepsy and other sequelae in asymptomatic neurocysticercosis.

Generic Name: Aldesleukin
Trade Name: Proleukin
Indication: Treatment of metastatic renal cell carcinoma.

Generic Name: Aldesleukin
Trade Name: Proleukin
Indication: Treatment of metastatic melanoma.

Generic Name: Alglucerase injection
Trade Name: Ceredase
Indication: For replacement therapy in patients with Gaucher's disease type I.

Generic Name: Alitretinoin
Trade Name: Panretin
Indication: Topical treatment of cutaneous lesions in patients with AIDS-related Kaposi's sarcoma.

Generic Name: Allopurinol sodium
Trade Name: Zyloprim for Injection
Indication: Management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy which causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy.

Generic Name: Alpha1-proteinase inhibitor (human)
Trade Name: Prolastin
Indication: For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state.

Generic Name: Altretamine
Trade Name: Hexalen
Indication: Treatment of advanced adenocarcinoma of the ovary.

Generic Name: Amifostine
Trade Name: Ethyol
Indication: For use as a chemoprotective agent for cisplatin in the treatment of advanced ovarian carcinoma.

Generic Name: Amifostine
Trade Name: Ethyol
Indication: Reduction of the incidence of moderate to severe xerostomia in patients undergoing post-operative radiation treatment for head and neck cancer.

Generic Name: Aminosalicylic acid
Trade Name: Paser Granules
Indication: Treatment of tuberculosis infections.

Generic Name: Amiodarone HCl
Trade Name: Cordarone
Indication: For the acute treatment and prophylaxis of life-threatening ventricular tachycardia or ventricular fibrillation.

Generic Name: Amphotericin B lipid complex
Trade Name: Abelcet
Indication: Treatment of invasive fungal infections.

Generic Name: Anagrelide
Trade Name: Agrylin
Indication: Treatment of essential thrombocythemia.

Generic Name: Antihemophilic factor (recombinant)
Trade Name: ReFacto
Indication: For the control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).

Generic Name: Antihemophilic factor (recombinant)
Trade Name: Kogenate
Indication: Prophylaxis and treatment of bleeding in individuals with hemophilia A or for prophylaxis when surgery is required in individuals with hemophilia A.

Generic Name: Antihemophilic factor/von Willebrand factor complex (human), dried, pasteurized
Trade Name: Humate-P
Indication: Treatment and prevention of bleeding in hemophilia A (classical hemophilia) in adult patients; and treatment of spontaneous and trauma-induced bleeding episodes in severe von Willebrand disease, and in mild and moderate von Willebrand disease where use of desmopressin is known or suspected to be inadequate in adult and pediatric patients.

Generic Name: Antithrombin III (human)
Trade Name: ATnativ
Indication: For the treatment of patients with hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or when they suffer from thromboembolism.

Generic Name: Antithrombin III (human)
Trade Name: Thrombate III
Indication: For replacement therapy in congenital deficiency of AT-III for prevention and treatment of thrombosis and pulmonary emboli.

Generic Name: Aprotinin
Trade Name: Trasylol
Indication: For prophylactic use to reduce perioperative blood loss and the homologous blood transfusion requirement in patients undergoing cardiopulmonary bypass surgery in the course of repeat coronary artery bypass graft surgery, and in selected cases of primary coronary artery bypass graft surgery where the risk of bleeding is especially high (impaired hemostasis) or where transfusion is unavailable or unacceptable.

Generic Name: Atovaquone
Trade Name: Mepron
Indication: Treatment of AIDS associated Pneumocystis Carinii Pneumonia.

Generic Name: Atovaquone
Trade Name: Mepron
Indication: Prevention of Pneumocystis carinii pneumonia (PCP) in high-risk, HIV-infected patients defined by a history of one or more episodes of PCP and/or a peripheral CD4+ (T4 helper/inducer) lymphocyte count less than or equal to 200/mm3.

Generic Name: Baclofen
Trade Name: Lioresal Intrathecal
Indication: Treatment of intractable spasticity caused by spinal cord injury, multiple sclerosis, and other spinal diseases (including spinal ischemia, spinal tumor, transverse myelitis, cervical spondylosis, and degenerative myelopathy).

Generic Name: Basiliximab
Trade Name: Simulect
Indication: Prophylaxis of solid organ rejection.

Generic Name: Benzoate and phenylacetate
Trade Name: Ucephan
Indication: For adjunctive therapy in the prevention and treatment of hyperammonemia in patients with urea cycle enzymopathy due to carbamylphosphate synthetase, ornithine, transcarbamylase, or argininosuccinate synthetase deficiency.

Generic Name: Beractant
Trade Name: Survanta intratracheal suspension
Indication: Treatment of neonatal respiratory distress syndrome.

Generic Name: Beractant
Trade Name: Survanta intratracheal suspension
Indication: Prevention of neonatal respiratory distress syndrome.

Generic Name: Betaine
Trade Name: Cystadane
Indication: Treatment of homocystinuria.

Generic Name: Bexarotene
Trade Name: Targretin
Indication: Treatment of cutaneous manifestations of cutaneous T-cell lymphoma in patients who are refractory to at least one prior systemic therapy.

Generic Name: Bleomycin sulfate
Trade Name: Blenoxane
Indication: Treatment of malignant pleural effusion.

Generic Name: Botulinum toxin type A
Trade Name: Botox
Indication: Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above).

Generic Name: Botulinum toxin type A
Trade Name: Botox
Indication: Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above).

Generic Name: Buffered intrathecal electrolyte/dextrose injection
Trade Name: Elliotts B Solution
Indication: For use as a diluent in the intrathecal administration of methotrexate and cytarabine for the prevention or treatment meningeal leukemia and lymphocytic lymphoma.

Generic Name: Busulfan
Trade Name: Busulfex
Indication: As preparative therapy in the treatment of malignancies with bone marrow transplantation.

Generic Name: Caffeine
Trade Name: Cafcit
Indication: Treatment of apnea of prematurity.

Generic Name: Calcitonin-human for injection
Trade Name: Cibacalcin
Indication: Treatment of symptomatic Paget's disease (osteitis deformans).

Generic Name: Calcium acetate
Trade Name: Phos-Lo
Indication: Treatment of hyperphosphatemia in end stage renal failure.

Generic Name: Chenodiol
Trade Name: Chenix
Indication: For patients with radiolucent stones in well opacifying gallbladders, in whom elective surgery would be undertaken except for the presence of increased surgical risk due to systemic disease or age.

Generic Name: Citric acid, glucono-delta-lactone and magnesium carbonate
Trade Name: Renacidin Irrigation
Indication: Treatment of renal and bladder calculi of the apatite or struvite variety.

Generic Name: Cladribine
Trade Name: Leustatin Injection
Indication: Treatment of hairy cell leukemia.

Generic Name: Clofazimine
Trade Name: Lamprene
Indication: Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum.

Generic Name: Clonidine
Trade Name: Duraclon
Indication: For continous epidural administration as adjunctive therapy with intraspinal opiates for the treatment of pain in cancer patients tolerant to, or unresponsive to, intraspinal opiates.

Generic Name: Coagulation factor IX
Trade Name: Mononine
Indication: Replacement treatment and prophylaxis of the hemorrhagic complications of hemophilia B.

Generic Name: Coagulation Factor IX (human)
Trade Name: AlphaNine
Indication: For use as replacement therapy in patients with hemophilia B for the prevention and control of bleeding episodes, and during surgery to correct defective hemostasis.

Generic Name: Coagulation Factor IX (recombinant)
Trade Name: BeneFix
Indication: Treatment of hemophilia B.

Generic Name: Coagulation factor VIIa (recombinant)
Trade Name: NovoSeven
Indication: Treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX.

Generic Name: Colfosceril palmitate, cetyl alcohol, tyloxapol
Trade Name: Exosurf Neonatal for Intratracheal Suspension
Indication: Prevention of hyaline membrane disease, also known as respiratory distress syndrome, in infants born at 32 weeks gestation or less.

Generic Name: Colfosceril palmitate, cetyl alcohol, tyloxapol
Trade Name: Exosurf Neonatal for Intratracheal Suspension
Indication: Treatment of established hyaline membrane disease at all gestational ages.

Generic Name: Corticorelin ovine triflutate
Trade Name: Acthrel
Indication: For use in differentiating pituitary and ectopic production of ACTH in patients with ACTH-dependent Cushings syndrome.

Generic Name: Cromolyn sodium
Trade Name: Gastrocrom
Indication: Treatment of mastocytosis.

Generic Name: Cromolyn sodium 4% ophthalmic solution
Trade Name: Opticrom 4% ophthalmic solution
Indication: Treatment of vernal keratoconjunctivitis.

Generic Name: Cysteamine
Trade Name: Cystagon
Indication: Treatment of nephropathic cystinosis.

Generic Name: Cytarabine liposomal
Trade Name: DepoCyt
Indication: Treatment of neoplastic meningitis.

Generic Name: Cytomegalovirus immune globulin (human)
Trade Name: CytoGam
Indication: Prevention or attenuation of primary cytomegalovirus disease in immunosuppressed recipients of organ transplants.


Please Click HERE for a comma-delimited list of the orphan drugs that you may add to your speller.

List From U.S. Federal Drug Administration (FDA)

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