FDA Approves Expanded Indication for Ferumoxytol

The US Food and Drug Administration (FDA) has approved AMAG Pharmaceuticals’ ferumoxytol (Feraheme) for an expanded indication to include all adult patients with iron deficiency anemia (IDA) who have an intolerance or unsatisfactory response to oral iron.

The approval essentially doubles the number of patients that can be treated with ferumoxytol. The FDA made its decision based on 2 phase 3 clinical trials that evaluated the therapy in comparison with iron sucrose and placebo, as well as ferric carboxymaltose injection.

“An expanded Feraheme label that includes all eligible adult IDA patients will double the addressable market opportunity estimated to be nearly a million patients per year for Feraheme and provide for future growth within this broader IDA market segment,” Nik Grund, the chief commercial officer at AMAG said in a statement.

Roughly 4.5 million Americans have IDA, all of which are now able to be treated with ferumoxytol. Ferumoxytol is currently protected under 7 patents, 6 of which are listed in the FDA’s Orange Book, the last of which will expire in June 2023.

"Iron deficiency anemia is a serious and under-treated health condition which negatively impacts the quality of life for millions of people, many of whom do not benefit from or cannot tolerate oral iron therapy,” Michael Auerbach, MD, a clinical professor at Georgetown University School of Medicine said in a statement. "Physicians now have a new option for patients who meet the broader ferumoxytol injection indication that can be administered in 15 minutes providing a gram of iron in two doses as few as three days apart."

AMAG submitted a New Drug Application (NDA) for the therapy in early August 2017, Ferumoxytol was previously approved by the FDA in 2009 for the treatment of IDA in adult patients with chronic kidney disease.

“The acceptance of our Feraheme label extension filing marks an important milestone in our effort to bring Feraheme to the more than 4.5 million Americans who have been diagnosed with IDA and suffer from its debilitating effects," Julie Krop, MD, the chief medical officer and senior vice president of clinical development and regulatory affairs at AMAG, said in a statement at the time of the NDA’s acceptance. “A significant proportion of these patients are women with IDA who suffer from gynecological issues, including abnormal uterine bleeding. We look forward to leveraging our deep relationships and expertise in women's health to bring a potential new treatment option to this underserved population.”

Ferumoxytol was studied in the FIRM trial, a phase 3 randomized, double-blind, noninferiority trial that compared the drug with ferric carboxymaltose injection (Injectafer, American Regent), which included an estimated 2,000 adults with IDA. Patients received either 2 doses of 510 mg ferumoxytol, or 2 doses of 750 mg ferric carboxymaltose over the course of 5 weeks.

The study met its primary endpoint, demonstrating noninferiority based on the composite of incidence of moderate to severe hypersensitivity reactions, including anaphylaxis, or moderate to severe hypotension.

As millions Americans having been diagnosed with IDA, “a significant proportion of these patients are women with iron deficiency anemia who suffer from gynecological issues, including abnormal uterine bleeding,” Krop said.

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