FDA Approves Accrufer for Treating Iron Deficiency in Children

In a significant advance for paediatric care, the United States Food and Drug Administration has approved the first prescription oral medicine to treat iron deficiency in children aged 10 years and older. The decision expands treatment options beyond dietary supplements and invasive therapies, addressing a widespread yet often under-diagnosed condition among school-age children and adolescents.

What the Approval Covers

The FDA has approved Accrufer, an oral capsule formulation of ferric maltol, for paediatric patients aged 10 and above with diagnosed iron deficiency. Until now, the drug had been authorised only for adults since 2019. With this extension, clinicians can prescribe a regulated, prescription-grade oral iron therapy to older children and teenagers requiring medical intervention rather than lifestyle advice alone.

Why Iron Deficiency in Children Matters

Iron deficiency remains the most common cause of anaemia worldwide. Low iron levels impair the body’s ability to produce haemoglobin, which transports oxygen in the blood. In children and adolescents, this can result in persistent fatigue, reduced concentration, delayed growth, and poorer academic performance. Causes include inadequate dietary intake, poor absorption, chronic blood loss, and heavy menstrual bleeding in adolescents.

Clinical Evidence Behind the Decision

The approval is based on results from the FORTIS clinical trial involving 24 patients aged 10 to 17 years. Participants received age-appropriate doses of Accrufer twice daily for 12 weeks. The study recorded an average increase in haemoglobin of 1.1 g/dL, a rise considered clinically meaningful and comparable to the effect of a single blood transfusion, without the need for injections or hospital-based procedures.

Imporatnt Facts for Exams

• Accrufer contains ferric maltol, an oral iron formulation.
• The FDA approval applies to children aged 10 years and above.
• Iron deficiency is the leading cause of anaemia globally.
• The FORTIS trial formed the evidence base for paediatric approval.

Safety Profile and Broader Implications

Accrufer is not suitable for all patients. It is contraindicated in children with iron overload disorders such as haemochromatosis, those receiving repeated blood transfusions, individuals with known allergies to its components, or patients experiencing active inflammatory bowel disease. Common side effects include gastrointestinal discomfort, constipation or diarrhoea, nausea, vomiting, gas, and darkened stools.

The approval offers families a convenient, non-invasive treatment option that can be administered at home, potentially reducing dependence on injections or transfusions. It also signals increased regulatory focus on paediatric nutrition-related disorders, an area historically under-recognised despite its long-term impact on health and development.