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Orphan Drugs Designated in 2003
I-S

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

INGN 201 (trade name: Advexin)
Treatment of head and neck cancer
Date designated: 1/27/2003

Introgen Therapeutics, Inc.
2250 Holcombe Blvd.
Houston, TX 77030 lactoferrin alpha
Treatment of graft versus host disease (GVHD)
Date designated: 8/20/2003

Also: for the prevention of GVHD
Date designated: 8/20/2003

Agennix, Inc.
8 Greenway Plaza, Suite 910
Houston, TX 77046

liposomal p-ethoxy growth receptor bound protein-2
Treatment of chronic myelogenous leukemia (CML)
Date designated: 12/5/2003

Interpath Pharmaceuticals, Inc.
2615 Hollymist
Houston, TX 77084

mafosfamide
Treatment of meningitis caused by cancer
Date designated: 1/21/2003

Baxter Oncology GmbH
Daimlerstrasse 40
60314 Frankfurt/Main

MaxAdFVIII
Treatment of classic hemophilia (hemophilia A)
Date designated: 3/3/2003

GenStar Therapeutics Corporation
10865 Altman Row, Suite 200
San Diego, CA 92121-1113

monarsen
Treatment of myasthenia gravis
Date designated: ?

Medica Venture Partners
11 Hamanofim St.
Ackerstein Towers
Building B, 10th Floor
Herzlia 46725, Israel

motexafin gadolinium (trade name: Xcytrin)
Treatment of metastatic brain tumors
Date designated: 1/27/2003

Pharmacyclics, Inc.
999 East Arques Avenue
Sunnyvale, CA 94085-4521

Rexin-G retroviral (trade name: Rexin-G)
Treatment of pancreatic cancer
Date designated: 8/15/2003

Epeius Biotechnologies Corporation
345 Pioneer Drive, 1803W
Glendale, CA 91203

myristoylated recombinant SCR1-3 of human complement receptor type I
Prevention of delayed graft function in solid organ transplant
Date designated: 5/21/2003

Adprotech, Ltd.
Chesterford Research Park
Little Chesterford
Near Cambridge
Essex, UK CB10 1XL

N-acetyl-sarcosyl-glycyl-L-valyl-D-alloisoleucyl-L-threonyl-L-norvalyl-L-isoleucyl-L-arginyl-L-prolylethylaminde acetate
Treatment of soft tissue sarcoma
Date designated: 12/9/2003

Abbott Laboratories
Pharmaceutical Products Division
200 Abbott Park Road
Abbott Park, IL 60064

niprisan (trade name: Hemoxin)
Treatment of sickle cell disease
Date designated: 8/15/2003

Xechem International, Inc.
New Brunswick Technology Center
100 Jersey Avenue, Building B, Suite 310
New Brunswick, NJ 08901

polyinosinic-polycytidilic acid (trade name: Poly-ICLC)
Treatment of flavivirus infections including West Nile, Japanese encephalitis, dengue, St. Louis encephalitis, yellow feer, Murray valley, and Banzai viruses
Date designated: 3/3/2003

Ribopharm, Inc.
3203 Cleveland Ave., NW
Washington, DC 20008-3450

protaxel
Treatment of ovarian cancer
Date designated: 5/21/2003

Biophysica, Inc.
3333 North Torrey Pines Court, Suite 100
La Jolla, CA 92037

recombinant adeno-associated virus alpha 1-antitrypsin vector (trade name: rAAV-AAT)
Treatment of alpha 1-antitrypsin deficiency
Date designated: 1/27/2003

Applied Genetic Technologies Corp.
12085 Research Drive, Suite 110
Alachua, FL 32615

recombinant human neutrophil inhibitor (hNE) (product name: DX-890)
Treatment of cystic fibrosis
Date designated: 12/9/2003

Dyax Corporation
300 Technology Square
Cambridge, MA 02139

recombinant inhibitor of human plasma kallikrein (product name: DX-88)
Treatment of angioedema
Date designated: 2/4/2003

Dyax Corporation
300 Technology Square
Cambridge, MA 02139

recombinant T-cell receptor (product name: VGI303)
Treatment of multiple sclerosis
Date designated: 5/2/2003

Virogenomics, Inc.
9020 SW Washington Square Road, Suite 380
Tigard, OR 97223-4332

repertaxin
Prevention of delayed graft function in solid organ transplant
Date designated: 1/27/2003

Dompe S.p.A.
Via Campo de Pile
67100 - L'Aquila - Italia

repository corticotropin or adrenocorticotropic hormone (trade name: H.P. Acthar Gel)
Treatment of infantile spasms (seizures)
Date designated: 5/21/2003

Questcor Pharmaceuticals, Inc.
3260 Whipple Road
Union City, CA 94587-1217

resiniferatoxin
Treatment of intractable (unable to be relieved) pain at end-stage disease
Date designated: 5/13/2003

Andrew J. Mannes, MD
Bldg 10/Rm 2N236
DASS/PPCS/NIDCR/NIH
10 Convent Drive
Bethesda, MD 20892

rhlGF-l/rhlGFBP-3 (trade name: SomatoKine)
Treatment of insulin resistance syndromes such as Type A, Type B, and Rabson-Mendenhall syndromes
Date designated: 12/9/2003

Insmed Inc.
4851 Lake Brooke Drive
Glen Allen, VA 23060

rhuMAb VEGF (bevacizumab) (trade name: Avastin)
Treatment of renal cell carcinoma
Date designated: 11/6/2003

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

SC-1 monoclonal antibody
Treatment of stomach tumors
Date designated: 11/12/2003

H3 Pharma, Inc.
666 Sherbrooke Street West, Suite 1400
Montreal, Quebec

SGN-30 (anti CD30 mAb)
Treatment of Hodgkin's disease
Date designated: 7/18/2003

Seattle Genetics, Inc.
21823 30th Drive, Southeast
Bothell, WA 98021

siplizumab
Treatment of T-cell lymphomas
Date designated: 7/15/2003

MedImmune Oncology, Inc.
35 W. Watkins Mill Road
Gaithersburg, MD 20878

sodium dichloroacetate
Antidote for systemic monochloroacetic acid poisoning
Date designated: 7/3/2003

EBD Group
6120 Paseo del Norte, Suite J2
Carlsbad, CA 92009

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