TABLE C-A1Orphan Drugs Relevant to Medicare Population (1983-2008 approvals) (99 drugs)

Exclusivity Start DateGeneric NameTrade NameIndication for Original Approvala
10/3/84Cromolyn sodium 4% ophthalmic solutionbOpticrom 4% ophthalmic solutionTreatment of vernal keratoconjunctivitis
11/30/84Naltrexone HClbReviaFor 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
8/30/85Potassium citratebUrocit-K(1) Prevention of calcium renal stones in patients with hypocitraturia. (2) Prevention of uric acid nephrolithiasis. (3) For avoidance of the complication of calcium stone formation in patients with uric lithiasis.
11/8/85Trientine HClSyprineTreatment of patients with Wilson's disease who are intolerant of, or inadequately responsive to, penicillamine
4/10/86LevocarnitinebCarnitorTreatment of genetic carnitine deficiency
12/15/86ClofazimineLampreneTreatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum
12/30/86Tranexamic acidcCyklokapronTreatment of patients with congenital coagulopathies who are undergoing surgical procedures (e.g., dental extractions)
3/19/87ZidovudinebRetrovir(1) Treatment of AIDS-related complex. (2) Treatment of AIDS.
8/11/88TioproninThiolaPrevention of cystine nephrolithiasis in patients with homozygous cystinuria
11/22/88Metronidazole (topical)bMetrogelTreatment of acne rosacea
5/2/89Mefloquine HClbLariam(1) Prophylaxis for Plasmodium falciparum malaria that is resistant to other available drugs. (2) Treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax.
5/25/89Rifampinb,cRifadin I.V.For antituberculosis treatment where use of the oral form of the drug is not feasible
6/5/89Selegiline HClbEldeprylAs an adjuvant to levodopa and carbidopa treatment of idiopathic Parkinson's disease (paralysis agitans), postencephalitic Parkinsonism, and symptomatic Parkinsonism
12/22/89Cromolyn sodiumGastrocromTreatment of mastocytosis
10/2/90Citric acid, glucono-delta-lactone and magnesium carbonatecRenacidin irrigationTreatment of renal and bladder calculi of the apatite or struvite variety
12/10/90Calcium acetatebPhos-loTreatment of hyperphosphatemia in end-stage renal failure
12/26/90AltretamineHexalenPalliative treatment of patients with persistent or recurrent ovarian cancer following first-line therapy
10/30/92Sotalol HClbBetapaceTreatment of life-threatening ventricular tachyarrhythmias
11/25/92AtovaquoneMepronFor the acute oral treatment of mild to moderate Pneumocystis carinii pneumonia in patients who are intolerant to trimethoprim-sulfamethoxazole
12/23/92RifabutinMycobutinPrevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection
7/23/93Interferon beta-1bc,dBetaseronIn ambulatory patients with relapsing-remitting multiple sclerosis to reduce the frequency of clinical exacerbations
9/10/93Megestrol acetatebMegaceTreatment of anorexia, cachexia, or an unexplained significant weight loss in patients with a diagnosis of acquired immune deficiency syndrome
9/23/93Lodoxamide tromethamineAlomide ophthalmic solutionTreatment of ocular disorders referred to by the terms vernal keratoconjunctivitis, vernal conjunctivitis, vernal keratitis
3/7/94Desmopressin acetatebN/ATreatment of patients with hemophilia A or von Willebrand's disease (type I) whose factor VIII coagulant activity level is greater than 5%
3/22/94Pilocarpineb,dSalagenTreatment of symptoms of xerostomia from salivary gland hypofunction caused by radiotherapy for cancer of the head and neck
5/31/94Rifampin, isoniazid, pyrazinamideRifaterFor the short-course treatment of tuberculosis
6/30/94Aminosalicylic acidPaser granulesTreatment of tuberculosis infections
7/29/94SulfadiazinebN/AToxoplasmosis, as adjunctive with pyrimethamine
8/15/94CysteamineCystagonTreatment of nephropathic cystinosis in adults and children
8/3/95Amiodarone HClbCordaroneFor initiation of treatment and prophylaxis of frequently recurring ventricular fibrillation and hemodynamically unstable ventricular tachycardia in patients refractory to other therapy
11/22/95Tretinoinb,dVesanoidInduction of remission in patients with acute promyelocytic leukemia who are refractory to or unable to tolerate anthracycline-based cytotoxic chemotherapeutic regimens
12/12/95Riluzoleb,dRilutekTreatment of patients with amyotrophic lateral sclerosis
4/30/96Sodium phenylbutyratedBuphenylAdjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase
5/17/96Interferon beta-1ac,eAvonexTreatment of relapsing forms of multiple sclerosis to slow the accumulation of physical disability and decrease the frequency of clinical exacerbations
5/17/96Allopurinol sodiumb,cAloprim for injectionManagement of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy that causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy
5/22/96OfloxacinbOcuflox ophthalmic solutionTreatment of bacterial corneal ulcers
6/11/96AlbendazoleAlbenza(1) Treatment of cystic hydatid disease of the liver, lung, and peritoneum, caused by the larval form of the dog tapeworm, Echinococcus granulosus. (2) Treatment of parenchymal neurocysticercosis due to active lesions caused by larval forms of the pork tapeworm, Taenia solium.
8/23/96Somatropin for injectionc,eSerostimTreatment of AIDS wasting or cachexia
9/6/96Midodrine HClbAmatineTreatment of symptomatic orthostatic hypotension
9/26/96Pentosan polysulfate sodiumElmironRelief of bladder pain or discomfort associated with interstitial cystitis
10/25/96BetaineCystadaneTreatment of homocystinuria
11/27/96Tizanidine HClbZanaflexTreatment of spasticity associated with multiple sclerosis and spinal cord injury
12/20/96Glatiramer acetatecCopaxoneFor reduction of the frequency of relapses in patients with relapsing-remitting multiple sclerosis
1/28/97Zinc acetateGalzinFor maintenance treatment of patients with Wilson's disease who have been initially treated with a chelating agent
3/14/97AnagrelidebAgrylinTreatment of patients with essential thrombocythemia
5/29/97ToremifenedFarestonTreatment of metastatic breast cancer in postmenopausal women with estrogen positive or receptor unknown tumors
12/10/97UrsodiolbUrsoTreatment of patients with primary biliary cirrhosis
2/25/98HydroxyureabDroxiaTo reduce the frequency of painful crises and to reduce the need for blood transfusions in adult patients with sickle cell anemia with recurrent moderate to severe painful crises
4/9/98SacrosidaseSucraidOral replacement therapy of the genetically determined sucrase deficiency
6/5/98Mafenide acetate solutionSulfamylon solutionFor use as an adjunctive topical antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds
6/22/98RifapentinePriftinTreatment of pulmonary tuberculosis
7/16/98ThalidomideThalomidAcute treatment of the cutaneous manifestations of moderate to severe erythema nodosum leprosum (ENL) and as maintenance therapy for prevention and suppression of the cutaneous manifestations of ENL recurrences
8/24/98LamotriginebLamictalAdjunctive treatment of Lennox-Gastaut syndrome in pediatric and adult patients
8/24/98Infliximabc,eRemicadeTreatment of moderately to severely active Crohn's disease for the reduction of signs and symptoms, in patients who have an inadequate response to conventional therapy; and treatment of patients with fistulizing Crohn's disease for reduction in the number of draining enterocutaneous fistula(s)
12/24/98ModafinilProvigilImprove wakefulness in patients with excessive daytime sleepiness associated with narcolepsy
2/2/99AlitretinoinPanretinTopical treatment of cutaneous lesions in patients with AIDS-related Kaposi's sarcoma
3/19/99Lidocaine patch 5%Lidoderm patchFor relief of allodynia (painful hypersensitivity) and chronic pain in postherpetic neuralgia
6/28/99Doxorubicin liposomec,dDoxilTreatment of metastatic carcinoma of the ovary in patients with disease that is refractory to both paclitaxel-and platinium-based chemotherapy regimens
10/21/99ExemestanedAromasinTreatment of advanced breast cancer in postmenopausal women whose disease has progressed following tamoxifen therapy
12/29/99BexaroteneTargretinTreatment of cutaneous manifestations of cutaneous T-cell lymphoma in patients who are refractory to at least one prior systemic therapy
5/10/01Imatinib mesylatedGleevecTreatment of patients with chronic myeloid leukemia (CML) in blast crisis, accelerated phase, or in chronic phase after failure of interferon-alpha therapy
8/28/01TopiramatedTopamaxAs adjunctive therapy in patients 2 years and older with seizures associated with Lennox-Gastaut syndrome
11/20/01BosentanTracleerTreatment of pulmonary arterial hypertension
1/18/02NitisinoneOrfadinAdjunctive therapy to dietary restriction of tyrosine and phenylalanine in treatment of hereditary tyrosinemia type 1
7/17/02OxybateXyremTreatment of cataplexy associated with narcolepsy
10/8/02Buprenorphine in combination with naloxoneSuboxoneTreatment of opioid dependence in patients 16 years of age or older
10/8/02Buprenorphine hydrochloridedSubutexTreatment of opioid dependence in patients 16 years of age or older
11/22/02NitazoxanideAliniaTreatment of diarrhea caused by Cryptosporidium parvum and Giardia lamblia
3/25/03PegvisomantcSomavertTreatment of acromegaly in patients who have had an inadequate response to surgery and/or radiation therapy and/or other medical therapies, or for whom these therapies are not appropriate
7/31/03MiglustatdZavescaTreatment of mild to moderate Type I Gaucher disease in adults for whom enzyme replacement therapy is not a therapeutic option
12/1/03Somatropin (r-DNA)c,eZorbtiveTreatment of short bowel syndrome in patients receiving specialized nutritional support
3/8/04CinacalcetdSensiparTreatment of hypercalcemia in patients with parathyroid carcinoma
4/20/04Apomorphine HClcApokynFor the acute, intermittent treatment of hypomobility, “off” episodes, associated with advanced Parkinson's disease
5/17/04TinidazoleTindamax(1) Treatment of giardiasis caused by G. duodenalis (also termed G. lamblia). (2) Treatment of intestinal amebiasis and amebic liver abcess caused by E. histolytica.
6/10/04GlutamineNutrestoreTreatment of short bowel syndrome in patients receiving specialized nutritional support when used in conjunction with a recombinant human growth hormone that is approved for this indication
8/12/05Quinine sulfateQualaquinTreatment of uncomplicated Plasmodium falciparum malaria
10/28/05Nelarabinec,dArranonTreatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens
11/2/05DeferasiroxExjadeTreatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age or older
12/20/05SorafenibNexavarTreatment of patients with advanced renal cell carcinoma
12/27/05LenalidomideRevlimidTreatment of patients with transfusion dependant anemia due to low or intermediate-1 risk myelodysplastic syndromes associated with a deletion 5 q cytogenetic abnormality with or without additional cytogenetic abnormalities
3/1/06Cetuximabc,d,eErbituxFor use in combination with radiation therapy, for the treatment of locally or regionally advanced squamous cell carcinoma of the head and neck (SCCHN) and for use as a single agent for the treatment of patients with recurrent or metastatic SCCHN for whom prior platinum-based therapy has failed
3/29/06Tacrolimusb,cPrografProphylaxis of organ rejection in patients receiving allogenic heart transplants
4/28/06Recombinant human acid alpha-glucosidasec,eMyozymeFor use in patients with Pompe disease (GAA deficiency)
5/2/06DecitabinecDacogenFor treatment of patients with myelodysplastic syndromes
6/28/06DasatinibdSprycel(1) Treatment of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia with resistance or intolerance to prior therapy. (2) Treatment of adults with CML with resistance or intolerance to prior therapy including imatinib.
7/24/06Idursulfasec,d,eElapraseIndicated for patients with Hunter syndrome (mucopolysaccharidosis II, MPS II)
10/6/06VorinostatZolinzaTreatment of cutaneous manifestations in patients with cutaneous T-cell lymphoma (CTCL) who have progressive, persistent, or recurrent disease on or following two systemic therapies
5/30/07TemsirolimuscToriselTreatment of advanced renal cell carcinoma
5/31/07Somatropinc,eNorditropinTreatment of short stature in patients with Noonan's syndrome
6/15/07AmbrisentanLetairisTreatment of pulmonary arterial hypertension (WHO group I) in patients with WHO class II or III symptoms to improve exercise capacity and delay clinical worsening
8/30/07LanreotidecSomatuline depotLong-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy
9/13/07RaloxifeneEvistaReduction in risk of invasive breast cancer in postmenopausal women with osteoporosis and reduction in risk of invasive breast cancer in postmenopausal women at high risk for invasive breast cancer
10/29/07NilotinibdTasignaFor the use for chronic phase (CP) and accelerated phase (AP) Philadelphia chromosome positive chronic myelogenous leukemia (CML) in adult patients resistant to or intolerant to prior therapy that included imatinib
12/13/07SapropterinKuvanIndicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-(BH4-) responsive phenylketonuria (PKU)
2/27/08Rilonaceptc,eArcalystTreatment of cryopyrin-assisted periodic syndromes (CAPS)
8/15/08TetrabenazineXenazineTreatment of chorea associated with Huntington's disease
9/12/08Immune globulin (human)c,eGamunexTreatment of chronic inflammatory demyelinating polyneuropathy
11/14/08RufinamideBanzelAdjunctive therapy of seizures associated with Lennox-Gastaut syndrome
11/20/08EltrombopagPromactaTreatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy

NOTE: CIAS1 = cold-induced autoinflammatory syndrome.


We excluded drugs that are covered under Medicare Part B or that are not relevant for the Medicare population (i.e., removed orphan approvals for a pediatric indication). The drugs are sorted by the exclusivity date (i.e., date of approval of orphan indication) for first orphan approval with a relevant indication. Drugs with multiple orphan designations often have exclusivity different dates associated with each approved indication. The text for some indications has been abbreviated.


These drugs are available in generic form.


These drugs have one of the following routes of administration: injection, intravenous, intramuscular, irrigation, or subcutaneous.


These drugs have one or more indications on the Social Security Compassionate Allowances List.


These are biologics, as opposed to chemical entities.

From: C, Medicare Part D Coverage and Reimbursement of Orphan Drugs

Cover of Rare Diseases and Orphan Products
Rare Diseases and Orphan Products: Accelerating Research and Development.
Institute of Medicine (US) Committee on Accelerating Rare Diseases Research and Orphan Product Development; Field MJ, Boat TF, editors.
Washington (DC): National Academies Press (US); 2010.
Copyright © 2010, National Academy of Sciences.

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