FDA approves rare blood disease treatment

The FDA has approved PharmaEssentia Corp.'s drug Besremi, a treatment for a rare blood disease called polycythemia vera. 

The agency said Nov. 12 that the disease affects about 6,200 people in the U.S. each year. It causes the overproduction of red blood cells, which thickens the blood, slowing blood flow and increasing the odds of blood clots. 

Besremi is the first FDA-approved drug for the disease that patients can take regardless of their treatment history, and the first interferon therapy approved specifically for the disease. 

The drug is believed to work by attaching to certain receptors in the body that set off a chain reaction, making the bone marrow reduce blood cell production, the FDA said. 

Besremi is taken by injection under the skin once every two weeks. If it reduces excess blood cells and maintains normal levels for at least a year, the dosing frequency can be reduced to once every four weeks. 

The effectiveness of Besremi was tested in a trial that lasted 7½ years. In the trial, 61 percent of patients had a complete hematological response, meaning they had a red blood cell volume of less than 45 percent without a recent phlebotomy, normal white cell counts and platelet counts, a normal spleen size and no blood clots.

The FDA warned that the drug carries some risks, including low white blood cell levels, low platelet levels, joint pain, upper airway infection and flu-like illness. 

Read the full news release here

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