LEXINGTON, Mass.—AMAG Pharmaceuticals announced that it has submitted a supplemental new drug application (sNDA) to the United States Food and Drug Administration (FDA) for Feraheme (ferumoxytol) Injection for Intravenous (IV) use.
The sNDA requests FDA approval to expand the indication for ferumoxytol beyond the current indication for the treatment of iron deficiency anemia (IDA) in adult patients with chronic kidney disease (CKD) to all adult patients with IDA who have failed or could not tolerate oral iron treatment. The application includes data from two well-controlled phase III clinical trials of more than 1,400 patients.
"This submission marks an important corporate milestone and represents the culmination of several years of work here at AMAG. The regulatory approval of Feraheme for a broader IDA patient population would expand our market opportunity in the existing U.S. IV iron market, beyond our current CKD indication," said William Heiden, president and chief executive officer of AMAG. "We believe that Feraheme, if approved for this indication, could provide a new treatment option for patients suffering from iron deficiency anemia who cannot tolerate or do not respond to oral iron therapy."
The sNDA submission is based on data from a global phase III program that evaluated the use of ferumoxytol in a broad range of adult IDA patients, all of whom had failed or could not tolerate oral iron treatment. More than 1,400 patients were enrolled in the two phase III clinical trials, IDA-301 (placebo comparator) and IDA-302 (active comparator). Both studies achieved their primary efficacy endpoints, with meaningful improvements in hemoglobin from baseline to the 35-day endpoint of the studies. Adverse events and serious adverse events associated with IV iron therapy, including hypersensitivity reactions, were reported in both studies. No new safety signals, outside of those described in the current Feraheme label, were observed with ferumoxytol treatment in these studies. These clinical trials also included patient-reported outcomes data as pre-specified secondary and exploratory endpoints. These outcomes endpoints, including quantitative measures of patients' fatigue and measures of quality of life, captured the negative pre-treatment impact anemia has on these patients' lives—and the significant improvement in these scores following a one gram course of therapy with ferumoxytol.