FDA accepts AMAG’s Feraheme sNDA for review

AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) today announced that the United States Food and Drug Administration (FDA) has accepted for review the company's supplemental new drug application (sNDA) for Feraheme® (ferumoxytol) Injection for Intravenous (IV) use, which was submitted to the FDA in December 2012. 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 adult patients with IDA who have failed or could not take oral iron treatment. Under the Prescription Drug User Fee Act (PDUFA) guidelines, the sNDA is subject to a 10-month review by the FDA. With the acceptance of the submission, the FDA has set October 21, 2013 as a target date for completion of their review.

"Now that our sNDA has been accepted, we look forward to working with the Agency to ensure a timely review of our submission," said William Heiden, president and chief executive officer of AMAG. "If approved for use in this patient population, Feraheme could provide an important new treatment option for patients suffering from iron deficiency anemia who cannot take 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 take oral iron treatment. More than 1,400 patients were enrolled in the two phase III clinical trials, known as IDA-301 (placebo comparator) and IDA-302 (active comparator). Both studies achieved their primary efficacy endpoints, with statistically significant improvements in hemoglobin from baseline to the 35-day endpoint of the studies. Adverse events and serious adverse events commonly associated with ferumoxytol and other IV iron therapies, including hypersensitivity reactions, were reported in both studies. No new safety signals, outside of those described in the current Feraheme® (ferumoxytol) 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 impact anemia has on these patients' lives pre-treatment - and the significant improvement in these scores following a one gram course of therapy with ferumoxytol. These data, and the safety and efficacy data from both IDA-301 and IDA-302 were presented at the 2012 Annual Meeting of the American Society of Hematology.