Orphan Drugs Approved in 2004

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 2004. Internet links for more information are provided where available.

acetylcysteine (trade name: Acetadote)
Intravenous treatment of moderate to severe acetaminophen overdose
Approved for marketing: 1/23/2004

Cumberland Pharmaceuticals, Inc.
209 10th Street South, Suite 332
Nashville, TN 37203

apomorphine hydrochloride injection (trade name: Apokyn)
Treatment of the on-off fluctuations associated with late-stage Parkinson's disease
Approved for marketing: 4/20/2004

Mylan Bertek Pharmaceuticals, Inc.
781 Chestnut Ridge Road
P.O. Box 4310
Morgantown, WV 26505

azacitidine (trade name: Vidaza)
Treatment of myelodysplastic syndromes
Approved for marketing: 3/19/2004

Pharmion Corporation
2525 28th Street
Boulder, CO 80301

cinacalcet (trade name: Sensipar)
Treatment of hypercalcemia in individuals with parathyroid carcinoma
Approved for marketing: 3/8/2004

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

clofarabine (trade name: Clolar)
Treatment of children 1 to 21 years old with acute lymphoblastic leukemia
Approved for marketing: 12/28/2004

Genzyme Oncology
500 Kendall Street
Cambridge, MA 02142

L-glutamine (trade name: NutreStore)
For use in the treatment of short bowel syndrome
Approved for marketing: 6/10/2004

Nutritional Restart Pharmaceutical
4364 South Alston Avenue
Durham, NC 27713

multivitamin infusion without Vitamin K (trade name: M.V.I.-12)
Prevention of vitamin deficiency in people receiving home parenteral nutrition and anticoagulant therapy
Approved for marketing: 9/9/2004

Mayne Pharma (USA) Inc.
650 From Road, Mack-Cali Centre II
Second Floor
Paramus, NJ, USA 07652

pemetrexed disodium (trade name: Alimta)
Treatment of malignant pleural mesothelioma (cancer of the linings of the lungs)
Approved for marketing: 1/23/2004

Eli Lilly and Company
Lilly Corporate Center
Indianapolis, IN 46285

pentetate calcium trisodium injection and pentetate zinc trisodium injection (trade names: Ca-DTPA and Zn-DTPA)
Treatment of internal contamination with plutonium, americium, or curium
Approved for marketing: 8/11/2004

Hameln Pharmaceuticals gmbh
Langes Feld 13, D-31789 Hameln

tinidazole (trade name: Tindamax)
Treatment of trichomoniasis, giardiasis, intestinal amebiasis and amebic liver abscess
Approved for marketing: 5/17/2004

Presutti Laboratories, Inc.
1607 N. Douglas Ave.
Arlington Heights, IL 60004