Positive Results Reported for Ferumoxytol (Feraheme) in Iron Deficiency Anemia
FDA review scheduled for October (Mar. 15)
Positive preliminary data have been reported from a single-arm, open-label extension study that evaluated the safety and efficacy of repeat dosing with ferumoxytol (Feraheme, Amag Pharmaceuticals) in patients with persistent or recurring iron deficiency anemia regardless of the underlying cause and a history of unsatisfactory oral iron therapy or in whom oral iron could not be used.
Patients were eligible to enroll in the extension study after they completed a double-blind, placebo-controlled trial that randomly assigned patients to receive either a 1-gram intravenous course of ferumoxytol or placebo. The results of the previous placebo-controlled study were presented at the American Society of Hematology annual meeting in December 2012.
A total of 337 patients received an initial course of ferumoxytol in the extension trial; of these patients, 151 had received placebo in the previous study. A total of 244 patients received a second course of therapy. The study’s primary efficacy endpoint was the mean change in hemoglobin from baseline to week 5 following the first course of ferumoxytol.
The 151 patients who received their first course of therapy with ferumoxytol achieved a statistically significant mean increase of 2.6 g/dL in hemoglobin from baseline to week 5. This change was consistent with the 2.7-g/dL increase in hemoglobin reported for ferumoxytol-treated patients in the previous placebo-controlled study.
The major secondary endpoint was the mean change in hemoglobin from baseline to week 5 for each subsequent course of ferumoxytol after the first treatment course. With repeat dosing, ferumoxytol demonstrated a statistically significant increase in mean hemoglobin from baseline to week 5, with a mean increase of 1.5 g/dL following the second course of treatment and a 1.1-g/dL increase following the third course of treatment. The mean hemoglobin level achieved by patients at week 5 following each of the first three treatment courses with ferumoxytol were comparable: 11.7 g/dL in the first course; 11.9 g/dL in the second course; and 11.4 g/dL in the third course.
In December 2012, a supplemental new drug application (sNDA) was submitted to the FDA for Feraheme (ferumoxytol) injection requesting agency approval to expand the indication for ferumoxytol beyond the current approved indication for the treatment of IDA in adult patients with chronic kidney disease to all adult patients with IDA who have failed or could not tolerate oral iron treatment. This sNDA is currently under review. The Prescription Drug User Fee Act target date is October 21, 2013.
Source: Amag Pharmaceuticals; March 15, 2013.