List of Orphan drugs: Part 1

I will like to bring to your vision, some of the orphan drugs which have been approved and marketed in the recent years.

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.

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

19. cetuximab (trade name: Erbitux)
For treatment of squamous cell cancer of the head and neck in patients who express epidermal growth factor receptor (EGFR). Cetuximab targets EGFR in the cancer cells and interferes with the cell growth, causing the cells to die.
Approved for marketing: 3/1/2006

ImClone Systems Incorporated

20. tacrolimus (trade name: Prograf)
For preventing organ rejection in patients receiving heart transplants

Astellas Pharma US, Inc.

21. ibuprofen lysine (trade name: NeoProfen)
For treatment of patent ductus arteriosus, a heart defect often occurring in premature babies.
Approved for marketing: 4/13/2006

Farmacon-IL, LLC

22. alpha-glucosidase (trade name: Myozyme)
For long-term enzyme replacement therapy in Pompe disease. This was the first treatment ever approved for the disorder.

Genzyme Corporation

23. decitabine (trade name: Dacogen)
For treatment of myelodysplastic syndrome (MDS), a disorder in which bone marrow that does not produce enough mature blood cells
Approved for marketing: 5/3/2006

MGI Pharma Inc.

24. infliximab (trade name: Remicade)
For treatment of Crohn’s disease in children ages birth to 16 years old
Approved for marketing: 5/19/2006

Centocor, Inc.

25. thalidomide (trade name: Thalomid)
Approved for marketing: 5/25/2006

26. lenalidomide (trade name: Revlimid)
Approved for marketing: 6/29/2006
Both for treatment of multiple myeloma, a disorder in which cancerous plasma cells in the blood form a tumor in the bone marrow, called myeloma; tumors in several sites is called multiple myeloma

Celgene Corporation

27. dasatinib (trade name: Sprycel)
Dasatinib was approved for two uses: treatment of chronic myelogenous leukemia and treatment of Philadelphia-positive acute lymphoblastic leukemia
Approved for marketing: 6/28/2006

Bristol-Myers Squibb Company

28. idursulfase (trade name: Elaprase)
For long-term enzyme replacement therapy in mucopolysaccharidosis II (MPS II, Hunter syndrome), an inherited storage disease
Approved for marketing: 7/24/2006

Shire plc

29. biocarbonate injustate (trade name: Normocarb HF)
For use in management of patients underoing continuous renal replacement therapy (CRRT) with hemofiltration
Approved for marketing: 7/26/2006

30. 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

31. 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

32. 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

33. 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

34. 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

35. 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

36. 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

37. 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