This is a list of the
orphan drugs (specialty drugs to treat rare disorders or conditions) approved by the
U.S. Food and Drug Administration (FDA) for marketing in the United States in 2007. Some are existing drugs with new approved uses, and some are completely new treatments.
(The list is alphabetical by trade name.)
1. Trade name: Alphanate
Generic name: antihemophilic factor (human)
Approved for surgical and/or invasive procedures in individuals with von Willebrand disease in whom desmopressin is either ineffective or contraindicated
Marketing approval date: 1/31/2007
Grifols Biologicals Inc.
2. Trade name: Ceprotin
Generic name: protein C concentrate
Approved for treatment of congenital Protein C deficiency to prevent and treat blood clots in the veins and purpura fulminans
Marketing approval date: 3/30/07
3. Trade name: CNJ-016
Generic name: vaccinia imune globulin (human) intravenous
Approved for treatment and/or modification of complications resulting from smallpox vaccination
Marketing approval date: This product was originally approved by the FDA in 2005; in 2007 it was approved for treating additional complications.
Cangene Corporation
4. Trade name: Curosurf
Generic name: pulmonary surfactant replacement, porcine (poractact alfa)
Approved for treatment of respiratory distress syndrome in premature infants
Marketing approval date: Curosurf, an existing drug, was approved for this use in 2007.
Dey Laboratories
5. Trade name: Doxil
Generic name: doxorubicin HCL liposome injection
Approved for use in combination with Velcade (bortezomib) for the treatment of individuals with multiple myeloma
Marketing approval date: 5/17/2007
Johnson & Johnson Pharmaceutical Research & Development
6. Trade name: Infasurf
Generic name: surface active extract of saline lavage of bovine lungs (calfactant)
Approved for treatment and prevention of respiratory failure due to pulmonary surfactant deficiency in preterm infants
Marketing approval date: Infasurf, an existing drug, was approved for this use in 2007.
Forest Pharmaceuticals, Inc.
7. Trade name: Kuvan
Generic name: sapropterin dihydrochloride
Approved for treatment of phenylketonuria
Marketing approval date: 12/13/07
8. Trade name: Letairis
Generic name: ambrisentan
Approved for treatment of pulmonary arterial hypertension in patients with specific symptoms to improve exercise capacity and delay worsening of the disease
Marketing approval date: 6/15/2007
9. Trade name: Norditropin
Generic name: somatropin
Approved for treatment of short stature in individuals with Noonan syndrome
Marketing approval date: 5/31/07
Approved for long-term treatment of children with growth failure due to inadequate secretion of endogenous growth hormone
Marketing approval date: Norditropin, an existing drug, was approved for this use in 2007.
Novo Nordisk Pharmaceuticals
10. Trade name: Nutropin Depot
Generic name: somatropin
Approved for long-term treatment of growth failure due to a lack of adequate endogenous growth hormone secretion, for once- or twice-a-month administration
Marketing approval date: Nutropin, an existing drug, was approved for this use in 2007.
Genentech, Inc.
11. Trade name: ReFacto
Generic name: antihemophilic factor (recombinant)
Approved for the control and prevention of bleeding episodes in individuals with hemophilia A (congenital factor VIII deficiency or classic hemophilia)
Marketing approval date: ReFacto, an existing drug, was approved for this use in 2007.
Wyeth Pharmaceuticals, Inc.
12. Trade name: Saizen
Generic name: somatropin
Approved for long-term treatment of children with growth failure due to inadequate secretion of endogenous growth hormone
Marketing approval date: Saizen, an existing drug, was approved for this use in 2007.
Serono Laboratories, Inc.
13. Trade name: Soliris
Generic name: eculizimab
Approved for treatment of paroxysmal nocturnal hemoglobinuria to reduce the breakdown of red blood cells
Marketing approval date: 3/16/07
14. Trade name: Supprelin LA
Generic name: histrelin
Approved for treatment of central precocious puberty
Marketing approval date: 5/3/07
Indevus Pharmaceuticals
15. Trade name: Somatuline Depot
Generic name: lanreotide
Approved for long-term treatment of individuals with acromegaly who have not responded well to or cannot be treated with surgery and/or radiation therapy
Marketing approval date: 8/30/07
16. Trade name: ThyroShield
Generic name: potassium iodide oral solution[/br]
Approved for use as a thyroid blocking agent in children exposed to radioactive iodine
Marketing approval date: ThyroShield, an existing drug, was approved for this use in 2007.
Fleming & Company Pharmaceuticals
17. Trade name: Torisel
Generic name: temsirolimus
Approved for treatment of advanced renal cell carcinoma (kidney cancer)
Marketing approval date: 5/30/07
Wyeth Pharmaceuticals, Inc.
18. Trade name: Totect
Generic name: dexrazoxane
Approved for treatment of extravasation (leaking under the skin) of intravenous anthracycline chemotherapy
Marketing approval date: 9/6/07
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