Orphan Drugs Approved in 2005

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 2005. Internet links for more information are provided where available.

desferasirox (trade name: Exjade)
Treatment of chronic transfusional hemosiderosis in patients aged two years and older. Reduces iron in transfused patients with thalassemia, sickle cell disease, and other rare anemias and myelodysplastic syndromes.
Approved for marketing: 11/2/05

Novartis Pharmaceuticals Corporation
One Health Plaza
East Hanover, NJ 07936-1080

fluocinolone acetonide intravitreal implant (trade name: Retisert)
Retisert is the first intravitreal drug implant approved for treatment of chronic noninfectious uveitis affecting the posterior segment of the eye.
Approved for marketing: 4/11/05

Bausch & Lomb
Offices worldwide

galsulfase (trade name: Naglazyme)
The first specific therapy approved for the treatment of mucopolysaccharidosis VI (MPS VI, also known as Maroteaux-Lamy syndrome)
Approved for marketing: 5/31/2005

BioMarin Pharmaceutical
105 Digital Drive
Novato, CA 94949

lenalidomide (trade name: Revlimid)
Revlimid is indicated for the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality. Revlimid, which is chemically related to thalidomide, will be available through a Revlimid Education and Prescribing Safety Program called RevAssist via contracted pharmacies.
Approved for marketing: 12/27/05

Celgene Corporation
86 Morris Avenue
Summit, NJ 07901

nelarabine (trade name: Arranon)
Treatment of adults and children with T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) whose disease has not responded to or has relapsed after at least 2 chemotherapy regimens. Arranon is the first drug to treat this limited population of patients (estimated at 500 individuals per year in the U.S.).
Approved for marketing: 10/31/05

GlaxoSmithKline US
Offices throughout the United States
Customer Response Center: 1-888-825-5249

sodium phenylacetate and sodium benzoate (trade name: Ammonul)
Adjunctive therapy in the treatment of acute hyperammonemia and associated encephalopathy in patients with urea cycle enzyme deficiencies
Approved for marketing: 2/17/2005

Ucyclyd Pharma
8125 N. Hayden Road
Scottsdale, AZ 85258

temozolomide (trade name: Temodar)
Treatment of adult patients with newly-diagnosed glioblastoma multiforme concomitantly with radiotherapy and then as maintenance treatment. Temodar was previously approved to treat refractory anaplastic astrocytoma.
Approved for marketing: 3/15/2005

Schering-Plough Research Institute
2000 Galloping Hill Rd.
Kenilworth, NJ 07033

vaccinia immune globulin intravenous (human) (VIGIV)
VIGIV is a specialty antibody made from human plasma. It is to be used in treating and preventing a type of severe reaction that may be brought on by administration of the smallpox vaccine. It was developed in partnership with the U.S. Department of Defense Joint Vaccine Acquisition Program for use as a biological defense vaccine.
Approved for marketing: 2/18/05

DynPort Vaccine Company LLC, A CSC Company
64 Thomas Johnson Drive
Frederick, Maryland 21702

Last updated 12/30/05