Orphan Drugs Approved in 2003

Some are first drugs to treat rare conditions

These orphan drugs (specialty drugs to treat rare disorders or conditions) were approved by the U.S. Food and Drug Administration (FDA) for marketing in the U.S. in 2003. Internet links for more information are provided where available.

bortezomib (trade name: Velcade)
Treatment of multiple myeloma
Approved for marketing: 5/13/2003

Millennium Pharmaceuticals, Inc.
75 Sidney Street
Cambridge, MA 02139

botulism immune globulin (trade name: BabyBIG)
Treatment of infant botulism
Approved for marketing: 10/23/2003

California Department of Health Services
2151 Berkeley Way
Berkeley, CA 94701-1011

alpha-galactosidase (trade name: Fabrazyme)
Treatment of Fabry disease
Approved for marketing: 4/24/2003

Genzyme Corporation
One Kendall Square
Cambridge, MA 02139-1562

iron(III)-hexacyanoferrate(II) (trade name: Radiogardase)
Treatment of patients with known or suspected internal contamination with cesium or thallium
Approved for marketing: 10/2/2003

Heyl Chemisch-Pharmazeutische Fabrik GMBH & Co., KG
Goerzallee 253
D-14167 Berin, Federal Republic of Germany

laronidase (trade name: Aldurazyme)
Treatment of patients with mucopolysaccharidosis I (Hurler and Scheie diseases)
Approved for marketing: 4/30/2003

BioMarin Pharmaceutical, Inc.
371 Bel Marin Keys Boulevard, Suite 210
Novato, CA 94949-5608

miglustat (trade name: Zavesca)
Treatment of Gaucher disease
Approved for marketing: 7/31/2003

Actelion Pharmaceuticals Ltd.
Gewerbestrasse 16
CH - 4123 Allschwi

pegvisomant (trade name: Somavert)
Treatment of acromegaly
Approved for marketing: 3/25/2003

Sensus Corporation
98 San Jacinto Boulevard, Suite 430
Austin, TX 78701

porfimer (trade name: Photofrin)
Treatment of Barrett's esophagus
Approved for marketing: 8/1/2003

Axcan Scandipharm Inc.
22 Inverness Parkway, Suite 310
Birmingham, AL 35242

ribavirin (trade name: Rebetol)
Treatment of chronic hepatitis C in pediatric patients
Approved for marketing: 7/29/2003

Schering-Plough Corporation
2000 Galloping Hill Road
Kenilworth, NJ 07033

somatropin (r-DNA) (trade name: Serostim)
Treatment of short bowel syndrome
Approved for marketing: 12/1/2003

Serono Laboratories, Inc.
One Technology Place
Rockland, MA 02370

tositumomab (trade name: Bexxar)
Treatment of non-Hodgkin's B-cell lymphoma
Approved for marketing: 6/27/2003

Corixa Corporation
1124 Columbia Street, Suite 200
Seattle, WA 98104

A list of orphan drugs approved in 2002 is also available.