Orphan Drugs
Drug Name:Orphan Drugs
List Of Brands:
Indication Type Description:
Drug Interaction
Indication
Adverse Reaction
Contra-Indications
Drug Interaction:
ORPHAN DRUGS
(Ref. Drugs Facts and Comparison (1997) 51st Edition )
The Orphan Drug Act defines an orphan drug as a drug or biological product for the diagnosis ,treatment or prevention of a rare disease or condition.
A rare disease is one which affects <20,000 persons in the U.S. or one which affects
> 20,000 persons but for which no reasonable expectation that the cost of developing the drug and making it available will be recovered from sales of that drug in the U.S.
The FDA office of Orphan Products Development (OPD) provided information package that
includes an overview of the FDAs Orphan Drug Program, a brief description of the orphan
drug products grant program and a current list of designated orphan products.
OPDs information package also contains a directory sheet listing sources of information
about the treatment of rare diseases , patient organizations and the availability of orphan
drugs. Requests for the Rare diseases information directory , or the entire orphan drug
package , may be made by contacting OPD
Office of Orphan Products Development (HF-35)
5600 Fishers Lane
Rockville , MD 20857
(301) 827-3670 or (800) 300-7469
Fax- (301) 443 -4915
Some of the Orphan Drug Products are listed below
DRUG (TRADE NAME) PROPOSED USE SPONSOR
1.Aconiazide Tuberculosis Lincoin Diagnostics
P.O.Box 1128
Decatur IL,62525
217-877-2531
2. Altretamine Advanced ovarian U.S Bioscience
(Hexalen) adenocarcinoma 100 Front Street
Suite 400
West Conshohocken
PA 19428
800-447-3969
3.Aminosidine Mycobacterium avium Thomas P.Kanyok
(Gabbaromicina) complex, visceral PharmD
(Paramomycin ) leischmaniasis (kala azar)
tuberculosis
4. Ananain, comosain For enzymatic debridement Genzyme Corp
( Vianain) of severe burns One Kendall sq.
Cambidge, MA
02139
617-252-7500
5.Anaritide acetate Improvement of early renal Scios Nova Inc
(Auriculin) allograft function following 2450 Bayshore
transplatation,acute renal Pkwy
failure Mountain view
CA, 94043
415-966-1550
6.Antiepilepsirine Drug resistent generalized Childrens Hospital
tonic-clonic epilepsy (Columbus- OH)
children and adults
7 Antihemophillic factor Prophylaxis/treatment of Bayer Corp
(recombinant) bleeding in hemophilia A, Biological &
(Kogenate) for prophylaxiswhen Pharmaceutical Dn
surgery is required in 400 Morgan lane
these patients CT 06516
203-937-2000
8. Anti-tac humanized Prophylaxis of acute renal Hoffman-La Roche
(Zenepax) allograft rejection,
prevention of acute
graft -vs host disease
following bone marrow
transplantation
8.Antihemophillic factor Prophylaxis/treatment of Bayer Corp
(recombinant) bleeding in hemophilia A, Biological &
(Kogenate) for prophylaxis when surgery Pharmaceutical
is required in these patients Dvn.
400 Morgan lane
CT 06516
203-937-2000
9. Arcitumonab Diagnosis and localization Immunomedics
(99m Tc- labeled of primary, residual, recurrent 300 American Road
CEA serum) and medullary thyroid Morris Plains,
carcinoma NJ 07950
201-605-8200
10.Botulinum toxin Blepharospasm and Associated Synapse
type A strabismus associated Biologics,
(Botox) with dystonia in adults
(Dysport) (>12 years old), cervical Allergan Inc.
dystonia, dynamic 2525, Du pont Drive,
muscle contracture in Irvine,
pediatric cerebral plasy CA 92715- 9534
essential blepharospasm, 800-433-8871
synkinetic closure of the
eyelid associated with Porton products Ltd
VIII cranial nerve aberrant c/o Speywood
degeneration Pharma 30401
Agoura Road
Suite 102
Agoura Hills
CA 91301
818-879- 2200
11. Botulinum toxin Cervical dystonia Athena Neurosciences
type B 800 Gateway Blvd
South San Francisco
CA 94080
415-877-0900
12. Botulinum toxin Infant botulism CA Dept. Health
type F Services
13.Bromodeoxyuridine Radiation sensitizer in the NeoPharm
treatment of primary
brain tumor
14.Carbovir AIDS, symptomatic HIV GlaxoWelcome
infection and CD4 Five Moore Drive
count < 200/mm3 Research triangle Pk
NC 27709
919-248-2100
15. Chenodiol Radiolucent stones in Solvay
well opacifying gall 901 Sawyer Road
-bladders, where Marietta ,
elective surgery would GA 30062-2224
be undertaken except 770-578-9000
for the resence of
increased surgical risk
due to systemic disease
or age
16. Chimeric A2 Chrons disease Centocor Inc
(human murine) 200 Great Valley
1gG monoclonal Pkwy
anti-TNF antibody Malvern
(cA2) PA 19355
610-651-6000
17.Chimeric M-T412 Multiple Sclerosis Centor Inc.
( human murine)
1gG monoclonal
anti-CD4
18.Chimeric (murine Non-Hodgkins B-cell IDEC Pharma
variable, human lymphoma 11099
constant) Mab (C2B8) N.Torrey Prince
to CD20 Road # 160
La Jolla CA 92037
619-458- 0600
19.Chondroitinase Patients undrrgoing Storz Opthalmics
viretomy 3365 Tree Court
Industrial
St.Louis
MO 63122-6694
314-225-5051
20.Clostridial Advanced (involutional L.Hurst, MD & M.
collagenase or residual stage) Badalaemente,
Deputyrens disease Ph.D
21.Defibrotide Thrombotic thrombocytopenic Crinos
purpura international
22.Dehydrex Recurrent corneal Hollies Laboratories
erosion unresponsive to Inc
conventionaltherapy 30 Forest Notch
Cohasset
MA 02025-1198
617-383-0741
23.Desmopression Mild hemophilia A and Rhone-Poulenc Rorer
acetate Von Willebrand disease Pharmaceutical Inc.
500- Arcola Road
P.O. Box 1200
Collegeville
PA 19426
610-454-8000
24. Dibromodulcitol Recuurent invasive or Biopharmaceuticals
metastatic squamous
cervical carcinoma
25.Dihydrotestosterone Weight loss in AIDS Unimed Pharma
Androgel-DHT patients with HIV 2150 E Lake
associated wasting Cook Road Buffalo Grove
IL 60089
800-541-3492
Indication:
DRUG (TRADE NAME) PROPOSED USE SPONSOR
26. Disodium clodronate Hypercalcemia of Discovery Experimental
malignancy & Development Inc.
29949 SR 54 West
Wesley Chapel
FL 33543
813-973-7200
27. Disodium silbinium Hepatic intoxification Pharmaquest Corp
dihemisuccinate of Amanita phalodies 4470 Redwood HZwy
(Legalon) (mushroom poisioning) San Rafeal
CA 94903
415-491-6460
28. DMP 777 Therapeutic mangmt Genetech Inc
of patients with lung 460 Point San
disease antributable Bruno Buvd.
to cystic fibrosis San Francisco
CA 94080
415-225-1000
29. Eflornithine HCl Trypanosoma brucei Marion Merrel Dow
(Ornidyl) gambiense
(sleeping sickness)
30.Elcatonin Intrathecaal treatment Innapharma
of intractrable pain
31.Epoprostenol Primary pulmonary Glaxo-Wellcome
hypertension, Five Moore Drive
replacement of Research triangle
heparin in patients Park, NC 27709
requiring hemodialysis 919-248-2100
in increased risk of
hemorrhage
32. Etiocholanedione Aplastic anemia, SuperGen
Prader- Willi syndrome
33.FIAU Adjunctive treatment of Oclassen Pharma inc
chronic active Hepatitis B 100 Pelican Way
San Rafeal
CA 94901
415- 258-4500
34. Flumecinol hyperbilirubinemia in Farmacon Inc
newborns unresponsive 90 Grove Street
Suite 109
Ridgefield
CT 06877-4118
203-431-9989
35.Fluorouracil With interferon alpha-2a Hoffman- LaRoche
(Adrucil) for esophageal/advanced Roche laboratories
colrectal carcinoma with 340 kingsland street
leucovorin for metastatic Nutley
adenocarcinoma of the NJ 07110-1199
colon and rectum 800-526-6367
36.Gallium nitrate Hypercalcemia of Solopak Pharma Inc.
injection malignancy 1845 Tonne Road
(Ganite) to phototherapy Elk Grove Village
IL 600007-5125
847-806-0080
37.Gamma-hydroxy Narcolepepsy Orphan Medical
butyrate 13911 Ridgedale
Drive
Minnetonika
612-513-6900
38.Gammlinolenic Juveneile rheumatoid Robert B. Zurier, MD
acid arthiritis
39.Ganaxolone Infantile spasm CoCensys Inc
213 Technology
Drive
Irvine CA 92718
714-453-0131
40.Gelsoin, Respiratory symptoms Biogen
recombinant of cystic fibrosis, acute 14 Cambrige Center
human and chronic respiratory Cambrige
symptoms of bronchiectasis MA 02142
617-679-2000
41.Gossypol Cancer of adrenal cortex Marcus M.Reidenberg
MD
42.Gasperimus Acute renal graft rejection Bristol- Meyer-Squibb
(Spanidin) episodes P.O.Box 4000
Princeton
NJ 08543-4000
609-252-4000
43.Halofantrine Mild to moderate malaria Smith Kline Beecham
(Halfan) caused by susceptible One Franklin Plaza
strains of Plasmodium falciparum P.O. Box 7929
and P vivax Philadelphia
PA 19103
215-751-4000
44.Heme arginate Symptomatic stage of acute Leiras Pharma Inc.
(Normosang) porphyria, myelodysplastic 2345 Waukegan Road
syndromes Suite N-135
Bonnockburn
IL 60015
45.Imciromab Detecting early necrosis as Centocor
pentetate an indication of rejection of
(Myoscint) orthotopic cardiac transplants
46.Interleukin-2 Alone and with inteferon Hoffman-La Roche
(Tecelukin) alfa-2a for metastatic Roche Laboratories
renal- cell carcinoma 340 Kingsland Street
and metstatic malignant Nutley NJ 07110-1199
melanoma 800-527-6367
47.Isobutyramide Sickle cell disease and Alpha Therapeutic
(Isobutyramide beta-thalassemia, beta- susan P.Perrine, MD
oral solution) hemoglobinopathies 5555 Valley Blvd
and beta-thassemia Los Angeles
syndromes CA 90032
213-225-2221
48.Leupeptin An adjunct to microsurgical Res Triangle Pharm
49.Leuteinizing With recombinant human Serono Labs Inc
hormone follicle stimulating hormone 100 Longwater Circle
(recombinant for chronic anovulation due Norwell MA 02061
human) to hypogonadrotropic 617-982-9000
hypgonadism
50.Melnoma cell Invasive melanoma Donald L Morton
vaccine peripheral nerve repair MD
Adverse Reaction:
DRUG (TRADE NAME) PROPOSED USE SPONSOR
51.Methylnaltraxone Chronic opiod-induced The University of
constipation unresponsive Chicago
to conventional therapy
52.Mitoguazone Diffuse non-Hodgkins ILEX oncology
lymphoma including
AIDS-related diffuse
non-Hodgkins lymphoma
53. Mitolactol Adjunct therapy in the Biopharmaceuticals
treatment of primary
brain tumors
54.Monolaurin Congenital primary Cellary Pharmaceuticals
(Glylorin) ichthyosis 371 Bel Marin Keye
Suite 210
Novato CA 94949
55.Myelin Multiple sclerosis Autoimmune Inc
128 Spring Street
Lexington MA 02173
617-860-0710
56.Nebacumab Gram negative bacteremia Centocor Inc
(Centoxin) that has progressed to 200 Great valley
endotoxin shock Pkwy
Malvern PA 19355
610-651-6000
57.Neutrophin-1 Motor neuron disease/ Arthur dale Ericsson
amyotrophic lateral MD
sclerosis
58.OM 401 Prophylactic treatment of Omex International
(Drepanol) sickle cell disease 6001- Savoy
Suite 110
Houstan TX 77036
713-975-8325
59.Oncorad Ov103 Ovarian cancer Cytogen
600 College Road
East
Princeton NJ 08540
609-987-8200
60.Orgotien for Familial amyotrophic Oxis International
injection lateral sclerosis 6040- N.Cutter
associated with a Circle, Suite 317
mutation of the gene Portland OR 97212
(chromosome 21q) for 503-283-3911
copper , zinc, superoxide
dismutase
61.Pegademase Enzyme replacement in Enzon Inc.
Bovine ADA deficiency in patients 40 Kingsbridge Rd
(Adagen) with severe combined Piscataway
immnodeficiency (SCD) NJ 08854- 3998
908-980-4500
62.PEG- gluco Chronic enzyme replacement Enzon Inc
cerebrosidase therapy in Gauchers dsease 40 Kingsbridge Rd
(Lysodase) in patients who are deficient Piscataway
in glucocerebrosidase NJ 08854- 3998
908-980-4500
63.PEG- interleukin-2 Primary immunodeficiency Chiron therapeutics
associated with high-risk 4560 Horton Street
patients Emeryville
CA 94608
800-244- 7668
64.Phenylalanine Hyperphenylalaninemia Ibex Tech
ammonia- lyase
(Phenylase)
65.Phosphocysteine Cytinosis Medea Research labs
200 Wilson Street
Port Jefferson
NY 11776
516-331-7718
66. Polifeprosan Malignant glioma Guilford Pharm
20 with carmustine
(Glidel)
67.Poloxamer 188 Sickle cell crisis, CytRX
(RheothRx severe burns 150 Technology phwy
Copolymer) requiring hospitalisation Norcross GA 30092
400-368-9500
68.Poloxamer 331 Initial therapy of CytYRX
(Protox) toxoplasmosis in
AIDS patients
69.Polymeric Sickle cell anemia Capmed USA
Oxygen P.O.Box 14
Bryn Mawr.
PA 19010
70.Protein islet prepn. Type I diabetic patients VivoRx
encapsulated who are already on
(BetaRx) immunosuppression
71.Porfiromycin Head and neck cancer OncoRx
72.Prednimustine Malignant non-Hodgkins Pharmacia
(Sterecyt) lymphoma & Upjon
P.O. Box 16529
Columbus
OH 43216-6529
614-764-8100
73.Protirelin Prevention of infant UCB Pharmaceticals
respiratory syndrome P.O. Box 4400
associated with prematurity Hampton
VA 23664-0410
804-851-4618
74. R-VIII SQ Long term or hospital treatment Pharmacia
(Refacto) hemophilia A or treatment of & Upjon
hemophilia A in connection with P.O. Box 16529
surgical procedures Columbus
OH 43216-6529
614-764-8100
75.Recombinant Cervical cancer Cantab Pharm Res
vaccina (human
papipillomavirus)
(TA-HPV)
Contra-Indications:
DRUG (TRADE NAME) PROPOSED USE SPONSOR
76.Relaxin recombinant, Progressive systemic Connective
human) sclerosis Therapeutics
77. Respiratory Syncyctical Prophylaxis of respiratory Medimmune Inc.
Virus Immune Globulin syncytial virus lower 35 West Watkins
(human) respiratory tract infections Mill Road
(Hypermune RSV) in infants and young Gaithersburg
(Respigam) children espicially ones MD 20878
at high risk of disease, 301-417-0770
treatment of RSV
lower respiratory tract
infections in hospitalised
infants and young children
78. Retionic acid 9-cis Acute promyelocyte Ligand pharma Inc
leukemia 9393 Towne centre
Drive
San Diego
CA 92121
619-535-3900
79.Retinon Squamous metaplasia of Hann Opthalmic
the ocular surface epithelia Marketing Services
(conjuctiva or cornea) with 163 Meetinghouse
mucus deficiency and Road
keratonization Duxbury
MA 02332
80. Ricin (blocked) Small lung cancer ImmunoGen
conjugated murine 148 Sidney Street
MCA (n901) Cambridge
MA 02139
617-661-9312
81.Roquinimex Prolongs time to relapse Pharmacia
(Linomide) in leukemia patients who & Upjohn
have undergone autologous P.O.Box 16529
bone marrow Columbus
transplatatoion OH 43216-6529
82. Satumonab Detection of ovarian Cytogen
Pendetide carcinoma 600 College Road
(Oncoscint East
CR/OV) Princeton
NJ 08540
609-987-8200
83.Secalciferol Familial hypophosphatemic Lemmon Co.
(Osteo-D) rickets 650 Cathill Road
Sellersville
PA 18960
215-256-8400
84.Sermorelin Adjuvant to levodopa/ Serono Labs Inc
acetate carbadopa in idoipathic 100 Longwater
(Geref) Parkinsons disease Circle
(paralysis agitans) Norwell
postcecephalitic MA 02061
parkinsonism/ 617-982-9000
symptomatic parkinsonism
85.Serratia Primary brain maligancies Cell technology
marcesecens 1668 Valtec Lane
extract Boulder
(polyribosomes CO 80301
(Imuvert) 303-790-0587
86.Sorivudine Herpes Zoster(shingles) Bristol- Myers Squibb
(BRAVAVIR) in immunocompromised P.O. Box 4000
patients Princeton
NJ 08543-4000
609-252-4000
87.Succimer Mercury intoxification, lead Bock Pharmacal Co.
(Chemet) poisoning in children , P.O. Box 419056
prevention of cynide kidney St.Louis
stones in patients with MO 63141-9056
homozygous cysturia who 314-579-0770
are prone to stone
development
88.Sulfadiazine With pyrimethamine for EON Labs Manf Inc
Toxoplasma gondi 227-15 North
encephalatis in patients Conduit Avenue
with or without AIDS Laurelton
NY 11413
718-276-8600
89.Sulfapyridine Dermatitis herpetiformis Jacobus Pharma Co
37 Cleveland
NJ 08540
609-921-7447
90. Superoxide Protection of donor organ Pharmacia-Chiron
dismutase tissue from damage or Partnership
(human) injury mediated by P.O. Box 16529
oxygen derived free Columbus
radicals generated OH 43216-6529
during the necessary 614-764-8100
periods of ischemia
(hypoxia, anoxia) and
especially reperfusion,
associated with the operative
procedure
91.Sysorb Pk Verototoxogenic E Coli Synsorb Biotech
infections
92. Tiraticol With levothyroxine to Macrofina Labs
(Triacana) suppress TSH in patients Lapha Labs
with differentiated thyroid
cancer intolerant of adequate
doses of levothyroxine alone
93. Trosulfan Ovatian Cancer Medac GmbH
(Ovastat ) C/o Princeton
Regulatory Assoc.
65 South Main St
Pennigton
NJ O8534
609-951-9596
94.Trimetrexate Advanced non-small cell U.S Bioscience
glucuronate carcinoma of the lung 100 Front street
(Neutrexin) Pneumocystis carini (PCP) Suite 400
in AIDS patients , metastatic West Consholhocken
carcinoma of head and neck PA 19428
(eg. buccal cavity, pharynx 800-447-3969
and larynx , metastatic colo-
rectal adenocarcinoma,
pancreatic adenocarcinoma
95.Trisaccharides Moderate to severe forms Chembiomed Ltd
A and B hemolytic disease of the P.O.Box 8050
( Biosynject) new born from placental Edmonton
transfer of antibodies against AB T6H4NP
blood groups A and B,
prevention of ABO medical
hemolytic reactions from
ABO -incompatable bone
marrow transplantatins
including kidney, heart, liver,
pancreas
96.Uridine -5- Cystic fibrosis to facilitate Inspire Pharma
triphosphate the removal of lung sceretions
in the treatment of patients
with primary ciliary dyskinesia
97. Urogastrone Accelration of corneal Chiron Vision
triphosphate epithelial regeneration and 500 Iolab drive
healing of stromal incisions Claremont
(corneal trnasplant surgery) CA 91711
909-624-2020
98.Valine, Hyperphenylalalninemia Leas Res Products
isoleucine
and leucine
99.Vasoactive Acute esophageal impaction Research Triangle
intestinal Pharmaceuticals
polypeptide 4364 S. Alston Ave
Durham
NC 27713
919-544-4029
100.Zinc acetate Wilsons Disease Lemmon
650 Cathill Road
sellersville
PA 18960
215-256-8400
