FDA Expands use of Ferric Maltol for Adolescents with Iron Deficiency

The FDA has expanded the indication of ferric maltol (Accrufer) capsules to include adolescents aged 10 years and older, making it the first prescription oral therapy approved for iron deficiency in pediatric patients. The drug was previously approved in 2019 for adults with iron deficiency.

Disease or Condition

Iron deficiency is the most common cause of anemia and occurs if there is not enough iron in the blood. Without enough iron, the body cannot make sufficient hemoglobin, a protein inside red blood cells that carries oxygen throughout the body. Iron deficiency anemia can result in symptoms such as fatigue, pale skin, and dizziness. Causes can include blood loss from gastrointestinal conditions, menstruation, food malabsorption, and insufficient iron in the diet.

Data Supporting Accrufer

The efficacy of Accrufer to treat iron deficiency in pediatric patients ages 10-17 was assessed in the FORTIS trial (NCT05126901). The trial treated 24 patients with age-based dosing of Accrufer twice daily, and showed a clinically meaningful average increase in hemoglobin of 1.1 g/dL at Week 12. This, on average, would be the expected increase in hemoglobin with one blood transfusion.

Safety Information

Patients should not take Accrufer if they are allergic to Accrufer or any of its excipients, have hemochromatosis and other iron overload syndromes, or receive repeated blood transfusions. In addition, patients should avoid Accrufer if they are experiencing an inflammatory bowel disease flare and should be aware of the risks of iron overload. The most common side effects of Accrufer are flatulence, diarrhea, constipation, discolored feces, abdominal pain, nausea, vomiting, and abdominal discomfort/distension.