List of Orphan Drugs - Part 2

Dear all,

This is continuation of my trailing blog which highlights orphan drugs approved during the period 2003-04, their indications and the pharmaceuticals which manufactures them.

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

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.

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

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

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

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

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.

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

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

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

bortezomib (trade name: Velcade)
Treatment of multiple myeloma
Approved for marketing: 5/13/2003
Millennium Pharmaceuticals, Inc.

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

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

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

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

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

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

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

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

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

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

ibritumomab (trade name: Zevalin)
For treatment of B-cell non-Hodgkin's lymphoma
Approved for marketing: 2/19/02
IDEC Pharmaceuticals

imatinib (trade name: Gleevec )
For treatment of gastrointestinal stromal tumors (had been approved 5/01 for treatment of chronic myeloid leukemia)
Approved for marketing: 2/1/02
Novartis

nitisinone (trade name: Orfadin)
For treatment of tyrosinemia type I
Approved for marketing: 1/18/02
Swedish Orphan AB Drottninggatan 98

oxybate (trade name: Xyrem )
For treatment of cataplexy, a form of narcolepsy
Approved for marketing: 7/17/02
Orphan Medical, Inc.

rasburicase (trade name: Elitek)
For treatment of malignancy-associated or chemotherapy-induced hyperuricemia
Approved for marketing: 7/12/02
Sanofi-Synthelabo

synthetic porcine secretin
For use in conjunction with diagnostic procedures for pancreatic disorders to increase pancreatic fluid secretion
Also approved for use in the diagnosis of gastrinoma associated with Zollinger-Ellison syndrome
Approved for marketing: 4/4/02
ChiRhoClin, Inc.

treprostinil (trade name: Remodulin)
For treatment of pulmonary arterial hypertension
Approved for marketing: 5/21/02
United Therapeutics