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Orphan Drugs Designated in 2003
C-I

From Mary Kugler, R.N., for About.com

Updated: December 29, 2003

About.com Health's Disease and Condition content is reviewed by our Medical Review Board

capsaicin
Treatment of painful HIV-associated neuropathy
Date designated: 5/2/2003

NeurogesX, Inc.
San Carlos Business Park
981F Industrial Road
San Carlos, CA 94070-4117

carboxypeptidase G2 (trade name: Voraxaze)
Treatment of patients at risk of methotrexate toxicity
Date designated: 8/19/2003

Protherics, Inc.
5214 Maryland Way, Suite 405
Brentwood, TN 37027

cholic acid (trade name: Falkochol)
Treatment of inborn errors of synthesis and metabolism of bile acid and cholesterol
Date designated: 7/18/2003

Dr. Falk Pharma GmbH
Leinenweberstrasse 5
Postfach 6529

cinacalcet
Treatment of hypercalcemia in patients with parathyroid carcinoma
Date designated: 5/12/2003

Amgen, Inc.
One Amgen Center Drive
Thousand Oaks, CA 91320-1799

combretastatin A4 phosphate
Treatment of thyroid cancer
Date designated: 7/23/2003

OXiGENE, Inc.
321 Arsenal Street
Watertown, MA 02472

conjugated bile acids (trade name: COBARTin)
Treatment of short bowel syndrome
Date designated: 7/18/2003

Jarrow Formulas, Inc.
1824 S. Robertson Blvd.
Los Angeles, CA 90035-4317

allogenic thymic tissue for transplantation
Treatment for complete DiGeorge syndrome
Date designated: 8/15/2003

Duke University Medical Center
Division of Pediatric Allergy & Immunology
Erwin Road
Durham, NC 27710

cyclosporin
Prevent rejection in people requiring lung transplant
Date designated: ?

Chiron Corporation
4560 Horton Street
Emeryville, CA 94608

debrase (trade name: Debridase)
Debridement (cleaning) of deep burns in hospitalized patients
Date designated: 8/20/2003

MediWound, Ltd.
42 Ha'Yarkon St.
Yavneh, Israel

defibrotide
Treatment of hepatic veno-occlusive disease
Date designated: 5/21/2003

Gentium SpA
Piazza XX Settembre, 2
22079 Villa Guardia (CO)

dehydroepiandrosterone (DHEA) (trade name: Fidelin)
Replacement therapy in people with adrenal insufficiency
Date designated: 8/19/2003

Paladin Labs, Inc.
6111 Royalmount Avenue, Suite 102
Montreal QC H4P 2T4
Canada

dextran 1
Treatment of cystic fibrosis
Date designated: 3/21/2003

BCY LifeSciences, Inc.
160 Eglinton Ave. East, Suite 600
Toronto, Ontario M4P 3B5

DHA-paclitaxel (trade name: Taxoprexin)
Treatment of cancer of the stomach or lower esophagus
Date designated: 5/1/2003

Protarga, Inc.
2200 Renaissance Boulevard, Suite 450
King of Prussia, PA 19406

diferuloylmethane
Treatment of cystic fibrosis
Date designated: 6/13/2003

Seer Pharmaceuticals, LLC
P.O. Box 138
Southport, CT 06890

diphenylcyclopenone
Treatment of chronic severe forms of alopecia areata
Date designated: 6/13/2003

Lloyd E. King Jr.
Vanderbilt University
1900 Patterson Street, Suite 104
Nashville, TN 37203

eculizumab
Treatment of paroxysmal nocturnal hemoglobinuria (PNH)
Date designated: 8/20/2003

Alexion Pharmaceuticals, Inc.
352 Knotter Drive
Cheshire, CT 06410

erlotinib hydrochloride (trade name: Tarceva)
Treatment of malignant glioma (brain cancer)
Date designated: 7/18/2003

Genentech, Inc.
1 DNA Way South
San Francisco, CA 94080-4990

Factor XIII homodimer, recombinant DNA origin
Treatment of congenital Factor XIII deficiency
Date designated: 5/21/2003

Also: Prevention of bleeding associated with congenital Factor XIII deficiency
Date designated: 11/6/2003

ZymoGenetics, Inc.
1201 Eastlake Avenue East
Seattle, WA 98102

ferric hexacyanoferrate(II)
Treatment of patients with known or suspected internal contamination with cesium or thallium
Date designated: 6/26/2003

Degussa AG
Weissfrauenstr. 9
D-60287 Frankfurt

HepeX-B
Prevention of hepatitis B reinfection in patients who have received a liver transplant
Date designated: 7/15/2003

XTL Biopharmaceuticals, Ltd.
Kiryat Weizmann
Science Pk, Bldg 3
POB 370, Rehovot 76100

human anti-tumor necrosis factor alpha monoclonal antibody
Treatment of uveitis
Date designated: 1/16/2003

Centocor, Inc.
200 Great Valley Parkway
Malvern, PA 19355-1307

human gammaglobulin
Treatment of idiopathic inflammatory myopathies
Date designated: 11/14/2003

Protein Therapeutics, Inc.
2761 N. Country Club Road, Suite 202
Tucson, AZ 85716

hydroxocobalamin (trade name: Cyanokit)
Treatment of acute cyanide poisoning
Date designated: ?

EMD Pharmaceuticals, Inc.
P.O. Box 13319
2311 Shannon Road, Suite 500
Durham, NC 27707

JE049 (trade name: Icatibant)
Treatment of angioedema
Date designated: 11/25/2003

Jerini AG
Invalidenstr. 130
10115 Berlin

IDN 6556
Treatment of patients undergoing solid organ transplantation
Date designated: 8/19/2003

Idun Pharmaceuticals, Inc.
9380 Judicial Drive
San Diego, CA 92121

imexon (trade name: Amplimexon)
Treatment of pancreatic cancer
Date designated: 11/1/2003

AmpliMed Corporation
2321 Camino La Zorrela
Tucson, AZ 85718

infliximab (trade name: Remicade)
Treatment of pediatric Crohn's disease and treatment of pediatric ulcerative colitis

Also: treatment of giant cell arteritis
Date designated: ?

Also: treatment of chronic sarcoidosis
Date designated: ?

Centocor, Inc.
200 Great Valley Parkway
Malvern, PA 19355-1307

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