Orphan Designations (updated April 25, 2018)
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Generic NameTrade NameDesignation DateDesignationOrphan Drug StatusFDA Approval StatusApproved IndicationMarketing Approval DateExclusivity End DateContact CompanyContact Address 1Contact Address 2Contact CityContact StateContact ZipContact CountryCF Grid KeyYear of designationLag, designation to approval
2
Diaziquonen/a1983-11-10
Treatment of primary brain malignancies (grade III and IV astrocytomas).
Designated/WithdrawnN/AN/A
Warner-Lambert Company
2800 Plymouth Rd., P.O. Box 1047
Ann ArborMichigan48106USA3831983
3
Alpha-1-antitrypsin (recombinant DNA Origin)
n/a1984-01-01
As supplementation therapy for alpha-1-antitrypsin deficiency in the ZZ phenotype population.
Designated/WithdrawnN/AN/AChiron Corporation4560 Horton StreetEmeryvilleCalifornia94608USA2831984
4
AltretamineHexalen1984-02-09
Treatment of advanced adenocarcinoma of the ovary.
Designated/Approved
Approved for Orphan Indication
Single agent palliative treatment of patients with persistent or recurrent ovarian cancer following first-line therapy with a cisplatin and/or alkylating agent based combination.
1990-12-261997-12-26
Medimmune Oncology, Inc.
One Tower Bridge
100 Front St., Suite 400
West Conshohocken
Pennsylvania
19428USA98319842512
5
Pentamidine isethionate
Pentam 300
1984-02-28
Treatment of Pneumocystis carinii pneumonia.
Designated/Approved
Approved for Orphan Indication
1984-10-161991-10-16Fujisawa USA, Inc.3 Parkway North CenterDeerfieldIllinois60015USA10831984231
6
Levocarnitine
Carnitor1984-02-28
Treatment of genetic carnitine deficiency.
Designated/Approved
Approved for Orphan Indication
1986-04-101993-04-10
Leadiant Biosciences, Inc.
9841 Washingtonian BlvdSuite 500
Gaithersburg
Maryland20878USA13841984772
7
Cromolyn sodium
Gastrocrom
1984-03-08Treatment of mastocytosis.Designated/Approved
Approved for Orphan Indication
1989-12-221996-12-22Fisons Corporation
755 Jefferson Rd., P.O. Box 1710
RochesterNew York14603USA118319842115
8
BacitracinAltracin1984-03-13
Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.
DesignatedN/AN/A
A. L. Laboratories, Inc.
One Executive Drive
P.O. Box 1399
Fort LeeNew Jersey7024USA12841984
9
Hemin
Panhematin
1984-03-16
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.
Designated/Approved
Approved for Orphan Indication
1983-07-201990-07-20
Abbott Laboratories
Diagnostics DivisionAbbott ParkIllinois60064USA6831984-240
10
Ethanolamine oleate
Ethamolin1984-03-22
Treatment of patients with esophageal varices that have recently bled, to prevent rebleeding.
Designated/Approved
Approved for Orphan Indication
1988-12-221995-12-22QOL Medical
4445 North Highway A1A, 241
Vero BeachFlorida32963USA148419841736
11
Botulinum toxin type A
Botox1984-03-22
Treatment of strabismus and blepharospasms
Designated/Approved
Approved for Orphan Indication
Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)
1989-12-291996-12-29Allergan, Inc.2525 Dupont Drive
P.O. Box 19534
IrvineCalifornia92713USA48319842108
12
Botulinum toxin type A
Botox1984-03-22
Treatment of strabismus and blepharospasms
Designated/Approved
Approved for Orphan Indication
Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)
1989-12-301996-12-30Allergan, Inc.2525 Dupont Drive
P.O. Box 19534
IrvineCalifornia92713USA48319842109
13
Epoprostenol
Flolan1984-03-29
Replacement of heparin in patients requiring hemodialysis and who are at risk of hemmorrage
Designated/WithdrawnN/AN/A
Glaxo Wellcome Inc.
5 Moore Drive
Research Triangle Park
North Carolina
27709USA7831984
14
Succimer
Chemet Capsules
1984-05-09
Treatment of lead poisoning in children.
Designated/Approved
Approved for Orphan Indication
1991-01-301998-01-30
Bock Pharmacal Company
P.O. Box 419056Saint LouisMissouri63141USA198419842457
15
Pegademase bovine
Adagen1984-05-29
For enzyme replacement therapy for ADA deficiency in patients with severe combined immunodeficiency.
Designated/Approved
Approved for Orphan Indication
1990-03-211997-03-21
Sigma-tau Pharmaceuticals, Inc.
9841 Washingtonian BlvdSuite 500
Gaithersburg
Maryland20878USA208419842122
16
Monooctanoin
Moctanin1984-05-30
For dissolution of cholesterol gallstones retained in the common bile duct.
Designated/Approved
Approved for Orphan Indication
1985-10-311992-10-31
Ethitek Pharmaceuticals, Inc.
7855 Gross Point Road, Unit L
SkokieIllinois60077USA24841984519
17
ClofazimineLamprene1984-06-11
Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum.
Designated/Approved
Approved for Orphan Indication
1986-12-151993-12-15
Novartis Pharmaceutical Corporation
59 Route 10
East Hanover
New Jersey7936USA26841984917
18
viloxazine HCL
Catatrol1984-06-11
Treatment of cataplexy and narcolepsy
Designated/WithdrawnN/AN/A
Stuart Pharmaceuticals
Division of ICI AmericasWilmingtonDelaware19897USA23841984
19
Levocarnitine
Carnitor1984-07-26
Treatment of primary and secondary carnitine deficiency of genetic origin.
Designated/Approved
Approved for Orphan Indication
Treatment of primary systemic carnitine deficiency. (approved 12/27/85) Treatment of secondary carnitine deficiency. (approved 12/16/92)
1992-12-161999-12-16
Leadiant Biosciences, Inc.
9841 Washingtonian BlvdSuite 500
Gaithersburg
Maryland20878USA158419843065
20
Iodine 131 6B-iodomethyl-19-norcholesterol
n/a1984-08-01For use in adrenal cortical imaging.DesignatedN/AN/ADavid E. Kuhl, M.D.
University of Michigan Medical Center/1500 E. Med.
B1G412 /0028 University Hospital
Ann ArborMichigan48109USA21841984
21
chenodiolChenix1984-09-21
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
Designated/Approved
Approved for Orphan Indication
1983-07-281990-07-28Solvay901 Sawyer RoadMariettaGeorgia30062USA33841984-421
22
Quinacrine hydrochloride
n/a1984-10-17
For prevention of recurrence of pneumothorax in patients at high risk of recurrence.
Designated/WithdrawnN/AN/ALyphomed, Inc.41841984
23
Spiramycin
Rovamycine
1984-10-17
For symptomatic relief and parasitic cure of chronic cryptosporidiosis in patients with immunodeficiency.
Designated/WithdrawnN/AN/A
Rhone-Poulenc Rorer Pharmaceuticals
500 Arcola RoadCollegeville
Pennsylvania
19426USA36841984
24
Cyproterone acetate
Androcur1984-10-26Treatment of severe hirsutism.Designated/WithdrawnN/AN/A
Berlex Laboratories, Inc.
300 Fairfield RoadWayneNew Jersey7470USA38841984
25
Epoprostenol
Cycloprostin
1984-10-29
Replacement of heparin in patients requiring hemodialysis and who are at increased risk of hemorrhage.
Designated/WithdrawnN/AN/AUpjohn Company301 Henrietta StreetKalamazooMichigan49001USA28841984
26
Pentamidine isethionate
n/a1984-10-29
Treatment of Pneumocystis carinii pneumonia.
DesignatedN/AN/A
Aventis Behring L.L.C.
1020 First Avenue
PO Box 61501
King of Prussia
Pennsylvania
19406USA37841984
27
Potassium citrate
Urocit-K1984-11-01
1. Prevention of calcium renal stones in patients with hypocitraturia 2. For avoidance of the complication of calcium stone formation in patients with uric lithiasis.
Designated/Approved
Approved for Orphan Indication
Prevention of recurring calcium conating stines associated with a deficiency in the urine.
1985-08-071992-08-07
University of Texas Health Science Center at Dallas
5323 Harry Hines BlvdDallasTexas75235USA96851984279
28
Potassium citrate
Urocit-K1984-11-01Prevention of uric acid nephrolithiasis.Designated/Approved
Approved for Orphan Indication
1985-08-301992-08-30
University of Texas Health Science Center at Dallas
5323 Harry Hines BlvdDallasTexas75235USA27841984302
29
Digoxin immune FAB (Ovine)
Digibind1984-11-01
Treatment of potentially life threatening digitalis intoxication in patients who are refractory to management by conventional therapy.
Designated/Approved
Approved for Orphan Indication
1986-04-221993-04-22
Glaxo Wellcome Inc.
5 Moore Drive
Research Triangle Park
North Carolina
27709USA25841984537
30
Teniposide
Vumon For Injection
1984-11-01
Treatment of refractory childhood acute lymphocytic leukemia.
Designated/Approved
Approved for Orphan Indication
Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.
1992-07-141999-07-14
Bristol-Myers Squibb Pharmaceutical Research Institute
5 Research Parkway
P.O. Box 5100
Wallingford
Connecticut
6492USA88319842812
31
L-5 Hydroxytrytophan
n/a1984-11-01
Treatment of postanoxic intention myoclonus.
DesignatedN/AN/A
Watson Laboratories, Inc.
33 Ralph AvenueP.O. Box 30CopiaqueNew York11726USA39841984
32
Urogastrone
n/a1984-11-01
For acceleration of corneal epithelial regeneration and healing of stromal incisions from corneal transplant surgery.
DesignatedN/AN/AChiron Vision500 Iolab DriveClaremontCalifornia91711USA16841984
33
Selegiline HCl
Eldepryl1984-11-07
As an adjuvant to levodopa and carbidopa treatment of idiopathic Parkinson's disease (paralysis agitans), postencephalitic Parkinsonism, and symptomatic Parkinsonism.
Designated/Approved
Approved for Orphan Indication
1989-06-051996-06-05
Somerset Pharmaceuticals, Inc.
400 Morris Ave., Suite 7sDenvilleNew Jersey7834USA58319841671
34
Antimelanoma Antibody XMMME-001-DTPA 111 Indium
Antimelanoma Antibody Xmmme-001-Dtpa 111 Indium
1984-11-14
Diagnostic use in imaging systemic and nodal melanoma metastasis.
Designated/WithdrawnN/AN/AXoma Corporation2910 Seventh StreetBerkeleyCalifornia94710USA34841984
35
Antimelanoma Antibody XMMME-001-RTA
Antimelanoma Antibody Xmmme-001-Rta
1984-11-14
Treatment of stage III melanoma not amenable to surgical resection.
Designated/WithdrawnN/AN/AXoma Corporation2910 Seventh StreetBerkeleyCalifornia94710USA35841984
36
Iobenguane Sulfate I 131
n/a1984-11-14
For use as a diagnostic adjunct in patients with pheochromocytoma.
Designated/Approved/Withdrawn
Adjunctive diagnostic agent in the localization of primary or metastatic pheochromocytomas.
N/A2001-03-25
University of Michigan
UH B1 H410/0028
1500 E. Medical Center Drive
Ann ArborMichigan48109USA22841984
37
Antithrombin III (human)
Thrombate Iii
1984-11-26
For replacement therapy in congenital deficiency of AT-III for prevention and treatment of thrombosis and pulmonary emboli.
Designated/Approved
Approved for Orphan Indication
1991-12-301998-12-30Bayer Corporation
Pharmaceutical Division, Biological Products
400 Morgan Lane
New Haven
Connecticut
6516USA408419842590
38
Alpha1-proteinase inhibitor (human)
Prolastin1984-12-07
For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state.
Designated/Approved
Approved for Orphan Indication
For chronic replacement therapy of individuals having congenital deficiency of alpha1- proteinase inhibitor with clinically demonstrable panacinar emphysema.
1987-12-021994-12-02Bayer Corporation
Pharmaceutical Division, Biological Products
400 Morgan Lane
New Haven
Connecticut
6516USA308419841090
39
AmsacrineAmsidyl1984-12-07Treatment of acute adult leukemia.Designated/WithdrawnN/AN/A
Warner-Lambert Company
2800 Plymouth RoadAnn ArborMichigan48106USA45841984
40
Sodium monomercaptoundecahydro-closo-dodecaborate
n/a1984-12-18
For use in conjunction with a thermal or epithermal neutron beam in boron nuclear capture therapy of glioblastoma multiforme.
Designated/WithdrawnN/AN/A
Theragenics Corporation
42841984
41
Trientine HCl
Syprine1984-12-24
Treatment of patients with Wilson's disease who are intolerant, or inadequately responsive to penicillamine.
Designated/Approved
Approved for Orphan Indication
1985-11-081992-11-08
Merck Sharp & Dohme Research
Division Of Merck And Company
West Point
Pennsylvania
19486USA48841984319
42
Protirelin injection
n/a1985-01-10
Treatment of amyotrophic lateral sclerosis.
Designated/WithdrawnN/AN/A
Abbott Laboratories
200 Abbott Park RoadAbbott ParkIllinoisUSA51841985
43
FlumecinolZixoryn1985-01-15
Treatment of hyperbilirubinemia in newborn infants unresponsive to phototherapy.
Designated/WithdrawnN/AN/AFarmacon, Inc.Post House, Suite 213
1720 Post Road East
Westport
Connecticut
6880USA29841985
44
factor XIII concentrate (human)
Corifact1985-01-16
Treatment of congenital factor XIII deficiency
Designated/Approved
Approved for Orphan Indication
For the routine prophylactic treatment of congenital factor XIII deficiency
2011-02-172018-02-17CSL Behring LLC1020 First Ave.
PO Box 61501
King of Prussia
Pennsylvania
19406USA448419859528
45
factor XIII concentrate (human)
Corifact1985-01-16
Treatment of congenital factor XIII deficiency
Designated/Approved
Approved for Orphan Indication
Peri-operative management of surgical bleeding in adult and pediatric patients with congenital Factor XIII deficiency.
2013-01-242020-01-24CSL Behring LLC1020 First Ave.
PO Box 61501
King of Prussia
Pennsylvania
19406USA4484198510235
46
physostigmine salicylatephysostigmine salicylate
n/a1985-01-16
Treatment of Friedreich's and other inherited ataxias.
Designated/WithdrawnN/AN/A
Forest Pharmaceuticals.3-.3-.30 6
150 East 58th StreetNew YorkNew York10155USA1831985
47
Gamma-hydroxybutyric acid
n/a1985-01-22
Treatment of narcolepsy and the auxiliary symptoms of cataplexy, sleep paralysis, hypnagogic hallucinations, and automatic behavior.
Designated/WithdrawnN/AN/A
Sigma Chemical Company
53841985
48
Levomethadyl acetate hydrochloride
Orlaam1985-01-24
Treatment of heroin addicts suitable for maintenance on opiate agonists.
Designated/Approved
Approved for Orphan Indication
For the management of opiate dependence.
1993-07-092000-07-09
Biodevelopment Corporation
8180 Greensboro Drive, Suite 1000
McLeanVirginia22102USA478419853088
49
Erwinia L-asparaginase
n/a1985-01-25
For use as an alternative to E. coli asparaginase in those situations where repeat courses of asparaginase therapy for acute lymphoblastic leukemia are required or where allergic reactions force the discontinuance of the E. coli preparation.
Designated/WithdrawnN/AN/ALyphomed, Inc.46841985
50
Antithrombin III (human)
Atnativ1985-02-08
For the treatment of patients with hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or when they suffer from thromboembolism.
Designated/Approved
Approved for Orphan Indication
1989-12-131996-12-13
Pharmacia & Upjohn AB
Lindhagensgatan 133StockholmSweden508419851769
51
Antipyrine Test
n/a1985-02-21
For use as an index of hepatic drug-metabolizing capacity.
Designated/WithdrawnN/AN/A
Upsher-Smith Laboratories, Inc
4905 23rd Avenue NorthMinneapolisMinnesota55447USA52841985
52
Epidermal growth factor (human)
n/a1985-03-06
For promotion of cutaneous wound healing in extreme burn treatment protocols.
Designated/WithdrawnN/AN/AEthicon, Inc.P.O. Box 151SomervilleNew Jersey8876USA54851985
53
Superoxide dismutase (human)
n/a1985-03-06
For protection of donor organ tissue from damage or injury mediated by oxygen-derived free radicals that are generated during the necessary periods of ischemia (hypoxia, anoxia), and especially reperfusion, associated with the operative procedure.
DesignatedN/AN/A
Pharmacia-Chiron Partnership
4560 Horton StreetEmeryvilleCalifornia94608USA59851985
54
Naltrexone HCl
Trexan1985-03-11
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.
Designated/Approved
Approved for Orphan Indication
1984-11-301991-11-30
DuPont Pharmaceuticals
E.I. Du Pont De Nemours & Co.
WilmingtonDelaware19898USA60851985-101
55
Alglucerase injection
Ceredase1985-03-11
For replacement therapy in patients with Gaucher's disease type I.
Designated/Approved
Approved for Orphan Indication
Long-term enzyme replacement therapy for children, adolescents and adult patients with a confirmed diagnosis of Type I Gaucher disease who exhibit signs and symptoms that are severe enough to result in one or more of the following conditions: a) moderate-to-severe anemia; b) thrombocytopenia with bleeding tendency; c) bone disease; d) significant hepatomegaly or splenomegaly.
1991-04-051998-04-05
Genzyme Corporation
One Kendall SquareCambridge
Massachusetts
2139USA558519852216
56
Digoxin immune fab(ovine)
Digidote1985-03-11
Treatment of life-threatening acute cardiac glycoside intoxication manifested by conduction disorders, ectopic ventricular activity and (in some cases) hyperkalemia.
DesignatedN/AN/A
Boehringer Mannheim Corp.
1301 Piccard DriveRockvilleMaryland20850USA56851985
57
Oxymorphone
Numorphan H.P.
1985-03-19
For relief of severe intractable pain in narcotic-tolerant patients.
DesignatedN/AN/A
DuPont Merck Pharmaceutical Company
P.O. Box 80027WilmingtonDelaware19880USA61851985
58
Tretinoinn/a1985-04-15
Treatment of squamous metaplasia of the ocular surface epithelia (conjunctiva and/or cornea) with mucous deficiency and keratinization.
DesignatedN/AN/A
Hannan Ophthalmic Marketing Services, Inc
34 Sherrill Rd.Marshfield
Massachusetts
2050USA57851985
59
calfactantInfasurf1985-06-07
Treatment and prevention of respiratory failure due to pulmonary surfactant deficiency in preterm infants.
Designated/Approved
Approved for Orphan Indication
Treatment and prevention of respiratory failure due to pulmonary surfactant deficiency in preterm infants.
1998-07-01N/AONY, Inc.1576 Sweet Home RoadAmherstNew York14228USA628519854772
60
Gancyclovirn/a1985-06-07
Treatment of severe human cytomegalovirus infections in specific immunosuppressed patient populations.
Designated/WithdrawnN/AN/A
Burroughs Wellcome Company
3030 Cornwallis Road
P.O. Box 12700
Research Triangle Park
North Carolina
27709USA58851985
61
AnagrelideAgrylin1985-06-11Treatment of polycythemia vera.DesignatedN/AN/A
Roberts Pharmaceutical Corp.
Meridian Center II
Four Industrial Way West
EatontownNew Jersey7724USA69851985
62
Prednimustine
Sterecyt1985-06-17
Treatment of malignant non-Hodgkin's lymphomas.
Designated/WithdrawnN/AN/APharmacia Inc.P.O. Box 16529ColumbusOhio43216USA66851985
63
Midodrine HCl
Amatine1985-06-21
Treatment of patients with symptomatic orthostatic hypotension.
Designated/Approved
Approved for Orphan Indication
Treatment of symptomatic orthostatic hypotension.
1996-09-062003-09-06Shire725 Chesterbrook BlvdWayne
Pennsylvania
19087USA758519854095
64
Antithrombin III (human) concentrate IV
Kybernin P1985-07-02
For prophylaxis and treatment of thromboembolic episodes in patients with genetic AT-III deficiency.
Designated/WithdrawnN/AN/ACSL Behring1020 First Ave.
PO Box 61501
King of Prussia
Pennsylvania
19406USA71851985
65
Defibrotiden/a1985-07-05
Treatment of thrombotic thrombocytopenic purpura.
DesignatedN/AN/ACrinos InternationalVia Belvedere 1
Villa Guardia, Italy
22079USA72851985
66
zidovudineRetrovir1985-07-17Treatment of AIDSDesignated/Approved
Approved for Orphan Indication
Management of certain adult patients with symptomatic HIV infection (AIDS and advanced ARC) who have a history of cytologically confirmed Pneumocytis carinii pneumonia (PCP) or an absolute CD4 (T4 helper/induccer) lymphocyte count of less than 200/mm in the peripheral blood before therapy is begun
1987-03-191994-03-19
Glaxo Wellcome Inc.
5 Moore Drive
Research Triangle Park
North Carolina
27709USA81851985610
67
ammonium 21-tungsto-9-antimoniate
n/a1985-07-17
Treatment of acquired immunodeficiency syndrome.
Designated/WithdrawnN/AN/A
Rhone-Poulenc Rorer Pharm.
500 Arcola RoadCollegeville
Pennsylvania
19426USA77851985
68
Cromolyn sodium 4% ophthalmic solution
Opticrom 4% Ophthalmic Solution
1985-07-24
Treatment of vernal keratoconjunctivitis.
Designated/Approved
Approved for Orphan Indication
1984-10-031991-10-03Fisons Corporation755 Jefferson Rd.
P.O. Box 1710
RochesterNew York14603USA82851985-294
69
Trimetrexate glucuronate
n/a1985-07-25
Treatment of pancreatic adenocarcinoma.
Designated/WithdrawnN/AN/A
Medimmune Oncology, Inc.
One MedImmune Way
Gaithersburg
Maryland20878USA83851985
70
Pentosan polysulfate sodium
Elmiron1985-08-07Treatment of interstitial cystitis.Designated/Approved
Approved for Orphan Indication
Relief of bladder pain or discomfort associated with interstitial cystitis.
1996-09-262003-09-26Alza Corporation950 Page Mill Rd.
PO Box 10950
Palo AltoCalifornia94303USA178419854068
71
ifosfamiden/a1985-08-07
Treatment of soft tissue and bone sarcomas
DesignatedN/AN/A
Bristol-Myers Squibb Company
P.O. Box 4000
Mail Stop D12-02
PrincetonNew Jersey8543USA85851985
72
Pentastarch
Pentaspan1985-08-28
As an adjunct in leukapheresis to improve the harvesting and increase the yield of leukocytes by centrifugal means.
Designated/Approved
Approved for Orphan Indication
1987-05-191994-05-19
DuPont Pharmaceuticals
E.I. Du Pont De Nemours & Co.
WilmingtonDelaware19898USA80851985629
73
tranexamic acid
Cyklokapron
1985-09-09
Treatment of hereditary angioneurotic edema
Designated/Approved/Withdrawn
Treatment of patients with hemophilia for short term use (2 to 8 days) before and after tooth extraction to reduce or prevent hemorrhage abd reduce the need for replacement therapy
N/A1993-12-30Pharmacia Inc.P.O. Box 16529ColumbusOhio43216USA88851985
74
Rifampin, isoniazid, pyrazinamide
Rifater1985-09-12
For the short-course treatment of tuberculosis.
Designated/Approved
Approved for Orphan Indication
Short-course treatment of tuberculosis.
1994-05-312001-05-31
Hoechst Marion Roussel
P.O. Box 9627Kansas CityMissouri64134USA658519853183
75
Epoprostenol
Flolan1985-09-25
Treatment of primary pulmonary hypertension.
Designated/Approved
Approved for Orphan Indication
Long-term intravenous treatment of primary pulmonary hypertension in NYHA Class III and Class IV patients.
1995-09-202002-09-20
Glaxo Wellcome Inc.
5 Moore Drive
Research Triangle Park
North Carolina
27709USA878519853647
76
Hydroxycobalamin/sodium thiosulfate
n/a1985-10-04
Treatment of severe acute cyanide poisoning.
Designated/WithdrawnN/AN/AHall, Alan H., M.D.Mile 5.0 Pass Creek RoadPO Box 24
Elk Mountain
Wyoming82324USA95851985
77
Methotrexate sodium
Methotrexate
1985-10-21Treatment of osteogenic sarcoma.Designated/Approved
Approved for Orphan Indication
For the use of high dose methotrexate with leucovorin rescue in patients with non-metastatic osteosarcoma who have undergone surgical resection or amputation for the primary tumor.
1988-04-071995-04-07
Lederle Laboratories
Division of American Cyanamid Company
401 N. Middletown Road
Pearl RiverNew York10965USA99851985899
78
Ganciclovir sodium
Cytovene1985-10-31
Treatment of cytomegalovirus retinitis in immunocompromised patients with AIDS.
Designated/Approved/Withdrawn
N/A1996-06-23Syntex (USA), Inc.3401 Hillview AvenuePalo AltoCalifornia94304USA79851985
79
Monoclonal antiendotoxin antibody XMMEn-0e5
n/a1985-11-04
Treatment of patients with gram-negative sepsis which has progressed to shock.
Designated/WithdrawnN/AN/APfizer, Inc.235 East 42nd StreetNew YorkNew York10017USA68851985
80
Zinc acetate
Galzin1985-11-06Treatment of Wilson's disease.Designated/Approved
Approved for Orphan Indication
For maintenance treatment of patients with Wilson's disease who have been initially treated with a chelating agent.
1997-01-282004-01-28Lemmon Company1510 Delp DriveKulpsville
Pennsylvania
19443USA1118519854101
81
MesnaMesnex1985-11-14
For use as a prophylactic agent in reducing the incidence of ifosfamide-induced hemorrhagic cystitis.
Designated/Approved
Approved for Orphan Indication
1988-12-301995-12-30
Degussa Corporation
65 Challenger Road
Ridgefield Park
New Jersey7660USA868519851142
82
Allopurinol riboside
n/a1985-12-04
1. Treatment of Chagas' disease 2. Treatment of cutaneous and visceral leishmaniasis
Designated/WithdrawnN/AN/A
Burroughs Wellcome Company
3030 Cornwallis Road
P.O. Box 12700
Research Triangle Park
North Carolina
27709USA106851985
83
Somatrem for injection
Protropin1985-12-09
1. Long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion (prevalence 15,000) 2. Treatment of short stature associated with Turner's syndrome (prevalence 8000).
Designated/Approved
Approved for Orphan Indication
1985-10-171992-10-17Genentech, Inc.1 DNA Way
South San Francisco
California94080USA107851985-53
84
RifampinRifadin I.V.1985-12-09
For antituberculosis treatment where use of the oral form of the drug is not feasible.
Designated/Approved
Approved for Orphan Indication
1989-05-251996-05-25
Hoechst Marion Roussel
P.O. Box 9627Kansas CityMissouri64134USA768519851263
85
Colchicinen/a1985-12-09
For arresting the progression of neurologic disability caused by chronic progressive multiple sclerosis.
Designated/WithdrawnN/AN/A
Pharmacontrol Corporation
661 Palisade Ave., P.O. Box 931
Englewood Cliffs
New Jersey7632USA110851985
86
Antithrombin III human
Antithrombin Iii Human
1986-01-02
Preventing or arresting episodes of thrombosis in patients with congenital AT-III deficiency and/or to prevent the occurrence of thrombosis in patients with AT-III deficiency who have undergone trauma or who are about to undergo surgery or parturition.
DesignatedN/AN/A
American National Red Cross
9312 Old Georgetown RoadBethesdaMaryland20814USA114851986
87
flunarizineSibelium1986-01-06Treatment of alternating hemiplegia.DesignatedN/AN/A
Janssen Research Foundation
P.O. Box 200
TitusvilleNew Jersey8560USA94851986
88
Guanethidine monosulfate
Ismelin1986-01-06
Treatment of moderate to severe reflex sympathetic dystrophy and causalgia.
Designated/WithdrawnN/AN/A
Novartis Pharmaceuticals Corporation
59 Route 10
East Hanover
New Jersey7936USA63851986
89
TioproninThiola1986-01-17
Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.
Designated/Approved
Approved for Orphan Indication
1988-08-111995-08-11
Pak, Charles Y.C. M.D.
The University of Texas Health Scienc Center at Da
5323 Harry Hines Boulevard
DallasTexas75235USA123861986937
90
Benzoate and phenylacetate
Ucephan1986-01-21
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.
Designated/Approved
Approved for Orphan Indication
1987-12-231994-12-23ImmunexImmunex2525 McGaw Avenue
P.O. Box 19791
IrvineCalifornia92623USA113851986701
91
Anti pan T lymphocyte monoclonal antibody
Anti-T Lymphocyte Immunotoxin Xmmly-H65-Rta
1986-01-29
1. For ex-vivo treatment to eliminate mature T cells from potential bone marrow grafts 2. For in-vivo treatment of bone marrow recipients to prevent graft rejection and graft versus host disease.
Designated/WithdrawnN/AN/AXoma Corporation2910 Seventh StreetBerkeleyCalifornia94710USA109851986
92
Beractant
Survanta Intratracheal Suspension
1986-02-05
Treatment of neonatal respiratory distress syndrome
Designated/Approved
Approved for Orphan Indication
Treatment of ("rescue") of premature infants with RDS confirmed by x-ray and requiring mechanical ventilation
1991-07-011998-07-01Ross Laboratories625 Cleveland AvenueColumbusOhio43215USA1088519861972
93
Beractant
Survanta Intratracheal Suspension
1986-02-05
Treatment of neonatal respiratory distress syndrome
Designated/Approved
Approved for Orphan Indication
Prevention of RDS (hyaline membrane disease) in premature infants less than 1250 grams birthweight or with evidence of surfactant deficiency
1991-07-021998-07-02Ross Laboratories625 Cleveland AvenueColumbusOhio43215USA1088519861973
94
Potassium citrate and citric acid
n/a1986-02-14
For the dissolution and control of uric acid and cysteine calculi in the urinary tract.
Designated/WithdrawnN/AN/A
Willen Drug Company
97851986
95
TerlipressinGlypressin1986-03-06
Treatment of bleeding esophageal varices.
DesignatedN/AN/A
Ferring Pharmaceuticals Inc.
100 Interpace Parkway
Parsippany-Troy Hills
New Jersey7054USA117851986
96
Etidronate disodium
Didronel1986-03-21
Treatment of hypercalcemia of malignancy inadequately managed by dietary modification and/or oral hydration.
Designated/Approved
Approved for Orphan Indication
1987-04-211994-04-21MGI Pharma, Inc.
5775 West Old Shakopee Rd.
Suite 100
Bloomington
Minnesota55437USA121851986396
97
Diethyldithiocarbamate
Imuthiol1986-04-03Treatment of AIDS.DesignatedN/AN/A
Connaught Laboratories
Route 611, P.O. Box 187Swiftwater
Pennsylvania
18370USA132861986
98
Epoetin alfaEpogen1986-04-10
Treatment of anemia associated with end stage renal disease.
Designated/Approved
Approved for Orphan Indication
Treatment of anemia associated with chronic renal failure, including patients on dialysis (end stage renal disesae) and patients not on dialysis
1989-06-011996-06-01Amgen, Inc.1840 Dehavilland Drive
Thousand Oaks
California91320USA1248619861148
99
Eflornithine HCl
Ornidyl1986-04-18
Treatment of Pneumocystis carinii pneumonia in AIDS patients.
Designated/WithdrawnN/AN/A
Marion Merrell Dow, Inc.
P.O. Box 9627Kansas CityMissouri64134USA136861986
100
Eflornithine HCl
Ornidyl1986-04-23
Treatment of Trypanosoma brucei gambiense infection (sleeping sickness).
Designated/Approved
Approved for Orphan Indication
1990-11-281997-11-28
Hoechst Marion Roussel
P.O. Box 9627Kansas CityMissouri64134USA1358619861680
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