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: Lamotrigine
Trade Name: Lamictal
Indication: Treatment of Lennox-Gastaut syndrome.
Generic Name: Lepirudin
Trade Name: Refluden
Indication: Treatment of heparin-associated thrombocytopenia type II.
Generic Name: Leucovorin
Trade Name: Leucovorin calcium
Indication: For use in combination with 5-fluorouracil for the treatment of metastatic colorectal cancer.
Generic Name: Leucovorin
Trade Name: Leucovorin calcium
Indication: For rescue use after high dose methotrexate therapy in the treatment of osteosarcoma.
Generic Name: Leuprolide acetate
Trade Name: Lupron Injection
Indication: Treatment of central precocious puberty.
Generic Name: Levocarnitine
Trade Name: Carnitor
Indication: Treatment of primary and secondary carnitine deficiency of genetic origin.
Generic Name: Levocarnitine
Trade Name: Carnitor
Indication: Treatment of genetic carnitine deficiency.
Generic Name: Levocarnitine
Trade Name: Carnitor
Indication: Treatment of manifestations of carnitine deficiency in patients with end stage renal disease who require dialysis.
Generic Name: Levomethadyl acetate hydrochloride
Trade Name: Orlaam
Indication: Treatment of heroin addicts suitable for maintenance on opiate agonists.
Generic Name: Lidocaine patch 5%
Trade Name: Lidoderm Patch
Indication: For relief of allodynia (painful hypersensitivity), and chronic pain in post-herpetic neuralgia.
Generic Name: Liothyronine sodium injection
Trade Name: Triostat
Indication: Treatment of myxedema coma/precoma.
Generic Name: Liposomal amphotericin B
Trade Name: AmBisome
Indication: Treatment of cryptococcal meningitis.
Generic Name: Liposomal amphotericin B
Trade Name: AmBisome
Indication: Treatment of visceral leishmaniasis.
Generic Name: Lodoxamide tromethamine
Trade Name: Alomide Ophthalmic Solution
Indication: Treatment of vernal keratoconjunctivitis.
Generic Name: Mafenide acetate solution
Trade Name: Sulfamylon solution
Indication: For use as an adjunctive topical antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds.
Generic Name: Mefloquine HCl
Trade Name: Lariam
Indication: Prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs.
Generic Name: Mefloquine HCl
Trade Name: Lariam
Indication: Treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax.
Generic Name: Megestrol acetate
Trade Name: Megace
Indication: Treatment of patients with anorexia, cachexia, or significant weight loss (=/>10% of baseline body weight) and confirmed diagnosis of AIDS.
Generic Name: Melphalan
Trade Name: Alkeran For Injection
Indication: Treatment of patients with multiple myeloma for whom oral therapy is inappropriate.
Generic Name: Mesna
Trade Name: Mesnex
Indication: For use as a prophylactic agent in reducing the incidence of ifosfamide-induced hemorrhagic cystitis.
Generic Name: Methotrexate sodium
Trade Name: Methotrexate
Indication: Treatment of osteogenic sarcoma.
Generic Name: Metronidazole (topical)
Trade Name: Metrogel
Indication: Treatment of acne rosacea.
Generic Name: Midodrine HCl
Trade Name: Amatine
Indication: Treatment of patients with symptomatic orthostatic hypotension.
Generic Name: Mitoxantrone
Trade Name: Novantrone
Indication: Treatment of hormone refractory prostate cancer.
Generic Name: Mitoxantrone HCl
Trade Name: Novantrone
Indication: Treatment of acute myelogenous leukemia, also referred to as acute nonlymphocytic leukemia.
Generic Name: Modafinil
Trade Name: Provigil
Indication: Treatment of excessive daytime sleepiness in narcolepsy.
Generic Name: Monoclonal antibody PM-81
Trade Name:
Indication: Adjunctive treatment of acute myelogenous leukemia.
Generic Name: Monooctanoin
Trade Name: Moctanin
Indication: For dissolution of cholesterol gallstones retained in the common bile duct.
Generic Name: Morphine sulfate concentrate (preservative free)
Trade Name: Infumorph
Indication: For use in microinfusion devices for intraspinal administration in the treatment of intractable chronic pain.
Generic Name: Nafarelin acetate
Trade Name: Synarel Nasal Solution
Indication: Treatment of central precocious puberty.
Generic Name: Naltrexone HCl
Trade Name: Trexan
Indication: For blockade of the pharmacological effects of exogenously administered opioids as an adjunct to the maintenance of the opioid-free state in detoxified formerly opioid-dependent individuals.
Generic Name: Nitric oxide
Trade Name: INOmax
Indication: Treatment of persistent pulmonary hypertension in the newborn.
Generic Name: Octreotide
Trade Name: Sandostatin LAR
Indication: Treatment of severe diarrhea and flushing associated with malignant carcinoid tumors.
Generic Name: Octreotide
Trade Name: Sandostatin LAR
Indication: Treatment of acromegaly.
Generic Name: Octreotide
Trade Name: Sandostatin LAR
Indication: Treatment of diarrhea associated with vasoactive intestinal peptide tumors (VIPoma).
Generic Name: Ofloxacin
Trade Name: Ocuflox Ophthalmic Solution
Indication: Treatment of bacterial corneal ulcers.
Generic Name: Oprelvekin
Trade Name: Neumega
Indication: Prevention of severe chemotherapy-induced thrombocytopenia.
Generic Name: Paclitaxel
Trade Name: Taxol
Indication: Treatment of AIDS-related Kaposi's sarcoma.
Generic Name: Pegademase bovine
Trade Name: Adagen
Indication: For enzyme replacement therapy for ADA deficiency in patients with severe combined immunodeficiency.
Generic Name: Pegaspargase
Trade Name: Oncaspar
Indication: Treatment of acute lymphocytic leukemia.
Generic Name: Pentamidine isethionate
Trade Name: Nebupent
Indication: Prevention of Pneumocystis carinii pneumonia in patients at high risk of developing this disease.
Generic Name: Pentamidine isethionate
Trade Name: Pentam 300
Indication: Treatment of Pneumocystis carinii pneumonia.
Generic Name: Pentastarch
Trade Name: Pentaspan
Indication: As an adjunct in leukapheresis to improve the harvesting and increase the yield of leukocytes by centrifugal means.
Generic Name: Pentosan polysulfate sodium
Trade Name: Elmiron
Indication: Treatment of interstitial cystitis.
Generic Name: Pentostatin for injection
Trade Name: Nipent
Indication: Treatment of hairy cell leukemia.
Generic Name: Pilocarpine
Trade Name: Salagen
Indication: Treatment of xerostomia induced by radiation therapy for head and neck cancer.
Generic Name: Pilocarpine HCl
Trade Name: Salagen
Indication: Treatment of xerostomia and keratoconjunctivitis sicca in Sjogren's syndrome patients.
Generic Name: Polifeprosan 20 with carmustine
Trade Name: Gliadel
Indication: Treatment of malignant glioma.
Generic Name: Porfimer sodium
Trade Name: Photofrin
Indication: For the photodynamic therapy of patients with primary or recurrent obstructing (either partially or completely) esophageal carcinoma.
Generic Name: Potassium citrate
Trade Name: Urocit-K
Indication: For avoidance of the complication of calcium stone formation in patients with uric lithiasis.
Generic Name: Potassium citrate
Trade Name: Urocit-K
Indication: Prevention of uric acid nephrolithiasis.
Generic Name: Potassium citrate
Trade Name: Urocit-K
Indication: Prevention of calcium renal stones in patients with hypocitraturia.
Generic Name: Pulmonary surfactant replacement, porcine
Trade Name: Curosurf
Indication: For the treatment and prevention of respiratory distress syndrome in premature infants.
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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