Ferric Carboxymaltose Injection Receives FDA Approval for Pediatric Patients With Iron Deficiency Anemia

The FDA has approved ferric carboxymaltose injection (Injectafer; Daiichi Sankyo and American Regent) for the treatment of children aged 1 year or older with iron deficiency anemia who are intolerant to oral iron or who have had an unsatisfactory response to oral iron.

The treatment was first approved in 2013 for adults as a 1500 mg course of treatment, administered as 2 doses of 750 mg each, separated by at least 7 days. For all patients weighing less than 50 kg, the recommended dosage is 15 mg/ per kg of body weight intravenously in 2 doses separated by at least 7 days.

“We are pleased to build on the effective and proven treatment of Injectafer for adult [iron deficiency anemia] patients with the approval of this new pediatric use,” said Linda Mundy, MD, chief medical officer at American Regent, in a press release. “More than 1.7 million adult patients have been treated with Injectafer in the US.”

In 2 randomized clinical studies, a total of 1775 patients were exposed to ferric carboxymaltose injection. Adverse effects (AEs) reported in 2% or more of patients treated with ferric carboxymaltose injection included nausea (7.2%), hypertension (4%), flushing (4%), injection site reactions (3%), erythema (3%), hypophosphatemia (2.1%), dizziness (2.1%), and vomiting (2%).

Furthermore, pooled data from 2 phase 3 studies were analyzed to compare rates of AEs in 2 parallel group studies, and AEs reported in 2% or more of patients included injection site reactions (4%) and injection site extravasation (2%).

Safety data in pediatric patients were evaluated in study 1VIT17044, which was a randomized, active-controlled study in which 40 patients received ferric carboxymaltose injection at 15 mg/kg to a maximum single dose of 750 mg on days 0 and 7, for a maximum total dose of 1500 mg. The study included 10 patients between the ages of 1 and 12 and 30 patients between the ages of 12 and 17.

According to the press release, 38 patients were evaluable for safety in the control arm and received an age-dependent formulation of oral ferrous sulfate for 28 days. The median age of patients who received ferric carboxymaltose injection was 14.5 years of age.

Additionally, AEs reported by 4% or more of patients treated with ferric carboxymaltose injection were hypophosphatemia (13%), injection site reactions (8%), rash (8%), headache (5%), and vomiting (5%). Some AEs have also been identified during post-approval use of ferric carboxymaltose injection, including cardiac disorders, administration site conditions, metabolism and nutrition disorders, musculoskeletal and connective tissue disorders, nervous system disorders, skin and subcutaneous tissue disorders, and fetal bradycardia.

“Pediatric health care providers now have an option for pediatric patients as young as 1 year of age with iron deficiency anemia who are intolerant to oral iron or who have had an unsatisfactory response to oral iron,” Mundy said in the press release.

REFERENCE

Injectafer (ferric carboxymaltose injection) Receives FDA Approval for the Treatment of Pediatric Patients with Iron Deficiency Anemia. News release. American Regent; December 15, 2021. Accessed December 17, 2021. https://americanregent.com/media/3203/final_pp-us-in-2750_injectafer-pediatric-press-release_2021-v3.pdf