FDA to Weigh Benefits and Risks of Amgen Clotting Drug NPLATE

By Anna Boyd
12:40, March 11th 2008
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FDA to Weigh Benefits and Risks of Amgen Clotting Drug NPLATE

A Food and Drug Administration panel will vote Wednesday on whether Amgen Inc.’ NPLATE drug to treat a rare blood clotting disorder provides enough benefit to counteract the side effects associated with its use.

NPLATE is intended to treat Idiopathic Thrombocytopenic Purpura (ITP), an autoimmune disorder characterized by very low levels of plateles leading to uncontrolled bleeding. Plateles are involved in clotting and are needed to stop bleeding, and NPLATE is designed to boost levels in a different manner than other treatment.

Amgen’s drug would treat chronic forms of ITP. To support the drug, Amgen submitted the results of two main clinical studies involving patients with ITP who failed other treatments. The FDA said the studies showed most patients treated with NPLATE had an improvement in blood-platelet levels. According to the medical officer reviewing NPLATE, the product was a “safe and effective alternative in raising the platelet count to levels where patients are no longer considered to be at increased risk for serious bleeding.”

However, the FDA also noted that 14 of 204 patients treated with NPLATE had dangerous blood clots. In addition, 8 percent of patients taking NPLATE developed a resistance to the drug, while only 4 percent of patients taking placebo showed the similar results. The FDA also expressed concerns about abnormal marrow growth and malignant tumors seen in some patients.

The FDA experts on Wednesday will decide whether the drug’s benefits outweigh its risks, and whether it should be approved on a limited basis, for example, for patients who have not responded to other drugs.

If approved, NPLATE’s label would warn of safety problems seen in company studies. In addition, the company would distribute additional guides on the risks to patients. Also, the company proposed studying irregular bone marrow growth and other adverse reactions in four follow-up studies.

ITP, which causes bruising and bleeding after minor injuries, affects about 200,000 people in the U.S., according to the Platelet Disorder Support Association.



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Tags: FDA, Amgen, NPLATE
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