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FDA Investigating Procrit and Epogen Following Clinical Trial Deaths

Sep 26, 2008 | Parker Waichman Alonso LLP

Epoetin alfa, an anemia drug sold in the U.S. as Procrit and Epogen, is being reviewed by federal regulators after patients in a German stroke study treated with the drug died at a higher rate than those administered a placebo.  The Food & Drug Administration (FDA) said it will be receiving more data about the German epoetin alfa trial in the next several weeks.  Once that data has been analyzed, the FDA will communicate its conclusions and recommendations regarding this drug to the public.

Procrit and Epogen are known as erythropoiesis-stimulating agent (ESA).  They are made by Amgen, but Procrit is sold by Johnson & Johnson subsidiary Ortho Biotech under a licensing agreement. ESAs are a bioengineered version of a natural protein made in the kidney that stimulates the bone marrow to produce more red blood cells.  Procrit and Epogen are approved in the U.S. to treat anemia in cancer patients undergoing chemotherapy.

The German study involved a version of epoetin alfa called Eprex, made by Johnson & Johnson, which is not marketed in the U.S. According to the FDA, Johnson & Johnson  made it aware of preliminary safety findings from a clinical trial conducted in Germany investigating the use of epoetin alfa to treat acute ischemic stroke.  Treatment of anemia was not a goal of the trial and most patients were not anemic.

According to the FDA, the clinical trial was a double-blind, placebo-controlled, multicenter investigation in 522 adult patients with an MRI-confirmed ischemic stroke in the area of the middle cerebral artery.  Patients were randomized to either receive treatment with a placebo or epoetin alfa administered as an intravenous dose of 40,000 units - a relatively high dose - daily for three days.  R-tPA, a medication used to help dissolve blood clots, and often used for acute strokes, was also used when clinically indicated.   The FDA said the goal of the clinical trial was to determine whether a  high dose of epoetin alfa administered for three days would improve the ability of patients to care for themselves after their strokes.

Over a period of ninety days after the start of the trial, 16 percent of the patients who received epoetin alfa died, compared to only 9 percent of patients in the placebo group.  Roughly half of all deaths in both groups occurred within the first seven days after starting the drug, with death from intracranial hemorrhage (bleeding within the brain) occurring among approximately 4 percent of epoetin alfa patients compared to 1 percent of patients in the placebo group.  

The FDA said that it is aware of other clinical trials investigating the use of epoetin alfa to improve the functional outcomes of patients after stroke.  The agency said that the finding of increased mortality in patients receiving epoetin alfa in the German trial suggests the need to closely monitor patients enrolled in these trials for adverse outcomes and to evaluate whether the potential benefits for enrolled patients outweigh the risks in these trials.

The FDA will work with the manufacturers of ESAs and other sponsors of clinical studies to evaluate the risks and benefits associated with the investigational uses of these ESA products as potential “neuroprotective agents.”