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Orphan Drugs Designated in 2003
Many new products now being tested

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

These drugs and treatments received orphan drug designation in 2003 from the U.S. Food and Drug Administration (FDA). The designation means that the drug developers may proceed with testing the drug in clinical trials, with the ultimate goal of receiving marketing approval. (See Orphan Drugs Approved in 2003 for a list of orphan drugs which received FDA marketing approval.)

Some of these drugs are completely new. Others are existing drugs seeking approval for a new use. Some are the first drug proposed to treat a particular condition; others would add to treatments already available. Some of the drugs are only known by their chemical names on this list. Internet links for more information are provided where available.

(+/-)-7-[3-(4-acetyl-3-methoxy-2-propylphenoxy)propoxy]-3,4-dihydro-8-propyl-2H-1-benzopyran-2-carboxylic acid (product name: Biomed 101)
Treatment of vascular leak syndrome caused by interleukin-2 therapy
Date designated: 4/21/2003

BioMedicines, Inc.
2000 Powell Street, Suite 1640
Emeryville, CA 94608

(R)-N-[2-(6-chloro-methoxy-1H-indol-3yl)propyl]acetamide
Treatment of neuroleptic-induced tardive dyskinesia in schizophrenia patients
Date designated: 7/3/2003

Phase 2 Discovery, Inc.
3130 Highland Ave., Third Floor
Cincinnati, OH 45219

1,1'-[1,4-phenylenebis(methylene)-bis-1,4,8,11-tetraazacyclotetradecan
For use with filgrastim to improve stem cell (bone marrow) transplantation
Date designated: 7/10/2003

AnorMED Inc.
200 - 20353 64th Avenue
Langley, BC
Canada V2Y 1N5

2',3',5'-tri-o-acetyluridine
Treatment of mitochondrial disease
Date designated: 1/3/2003

RepliGen Corporation
41 Seyon Street
Building 1, Suite 100
Waltham, MA 02453

2-(3-diethylaminopropyl)-8,8-dipropyl-2-azaspiro [4,5[ decan dimaleate (trade name: Atiprimod)
Treatment of multiple myeloma
Date designated: 12/2/2003

Callisto Pharmaceuticals, Inc.
2 Executive Drive, Suite 450
Sormerset, NJ 08873

INO-4995
Treatment of cystic fibrosis
Date designated: 8/15/2003

Inologic, Inc.
101 Elliott Ave. West
Seattle, WA 98119

TH9402 (trade name: Theralux Irradiation Device)
Treatment of chronic myelogenous leukemia (CML)
Date designated: 4/10/2003

Celmed BioSciences Inc.
2310 Alfred-Nobel Blvd.
Saint-Laurent (Quebec)
Canada H4S 2A4

5,5',5"-[Phosphinothioylidyne-tris(imino-2,1-ethanediyl)]tris[5-methylchelidoninium]trihydroide hexahydrochloride
Treatment of pancreatic cancer
Date designated: 8/20/2003

Now Pharm AG
241, route d'Arlon
L-1150

a-(3-aminophthalimido) (trade name: Actimid)
Treatment of multiple myeloma (See article: The Return of Thalidomide)
Date designated: 1/15/2003

Celgene Corporation
7 Powder Horn Drive
Warren, NJ 07059

a-galactosidase A (trade name: Plant-Produced Human a-Galactosidase)
Treatment of Fabry disease
Date designated: 1/21/2003

Large Scale Biology Corporation
3333 Vacaville Parkway, Suite 1000
Vacaville, CA 95688

ACA125
Treatment of epithelial ovarian cancer
Date designated: 11/25/2003

CellControl Biomedical Laboratories AG
Am Klopferspitz 19
82152 Martinsried (bei München)

alendronate (trade name: Fosamax)
Treatment of osteogenesis imperfecta in children 4 years of age and older
Date designated: 3/31/2003

Merck & Co., Inc.
126 East Lincoln Ave.
Rahway, NJ 07065-0900

alteplase (trade name: Activase)
Treatment of intraventricular hemorrhage
Date designated: 1/27/2003

Daniel F. Hanley, MD
Johns Hopkins University
600 N. Wolfe Street, Jefferson 1-109
Baltimore, MD 21287

AMG 531
Treatment of immune thrombocytopenic purpura (ITP)
Date designated: 3/27/2003

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

anti-CD23 IgG1, kappa monoclonal antibody
Treatment of chronic lymphocytic leukemia (CLL)
Date designated: 2/12/2003

IDEC Pharmaceuticals, Inc.
3030 Callan Road
San Diego, CA 92121

anti-CEA sheep-human chimeric monoclonal antibody
Treatment of pancreatic cancer
Date designated: 5/21/2003

KS Biomedix Ltd.
1 Occam Court
The Surrey Research Park
Guildford
Surrey
GU2 7HJ

anti-interferon-gamma Fab from goats
Treatment of immunologic corneal graft rejection
Date designated: ?

Advanced Biotherapy, Inc.
6355 Topanga Canyon Blvd., Suite 510
Woodland Hills, CA 91367

antisense 20-mer phosphorothioate oligonucleotide (trade name: GTI-2040)
Treatment of renal cell carcinoma
Date designated: 3/12/2003

Lorus Therapeutics, Inc.
2 Meridian Road
Toronto, Ontario
Canada, M9W 4Z7

arsenic trioxide (trade name: Trisenox)
Treatment of chronic lymphocytic leukemia (CLL)
Date designated: 5/13/2003

Also: Treatment of liver cancer
Date designated: 6/13/2003

Cell Therapeutics, Inc.
501 Elliott Avenue West, Suite 400
Seattle, WA 98119

atomoxetine hydrochloride (trade name: Strattera)
Treatment of Tourette syndrome
Date designated: 8/26/2003

Eli Lilly & Company
Lily Corporate Center
Indianapolis, IN 46285

baclofen
Treatment of dystonia
Date designated: 12/2/2003

Medtronic Neurological
710 Medtronic Parkway NE
Minneapolis, MN 55432-5604

bifidobacterium longum infantis
Treatment of pediatric Crohn's disease
Date designated: 1/16/2003

Alimentary Health
Guardwell, Kinsale
Co. Cork, Ireland

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