Orphan Drugs Approved by the FDA for Marketing in 2006

Some are first to treat a rare disorder

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 U.S. in 2006. Links for more information are provided where available.

1. 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

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

Astellas Pharma US, Inc.

3. 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
1720 Post Road East, Suite 213
Westport, CT 06880

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

• Read about FDA approval
• Read about EMEA approval

Genzyme Corporation

5. 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

• Read about FDA approval

MGI Pharma Inc.

6. 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.

7. thalidomide (trade name: Thalomid)
Approved for marketing: 5/25/2006
8. 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

• Read about the return of drug thalidomide
• Book review – Dark Remedy: The Impact of Thalidomide and Its Revival as a Vital Medicine

Celgene Corporation

9. 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

10. 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

• Read about FDA approval
• Read about the Phase II/III Elaprase study

Shire plc

11. 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

Dialysis Solutions, Inc.