Accrufer Becomes First FDA-Approved Prescription Iron Therapy for Children 10+

The FDA has approved Accrufer, an oral iron therapy from Shield Therapeutics, for the treatment of iron deficiency in pediatric patients aged 10 years and older. The approval expands the drug’s label beyond adults, for whom the therapy has been authorized since 2019, marking a new option for adolescents who require prescription iron therapy.

With the decision, Accrufer becomes the first and only prescription oral iron therapy approved for children aged 10 and above with iron deficiency.

Along with the expanded indication, the FDA has granted Accrufer three additional years of data exclusivity, extending exclusivity until December 19, 2028, alongside existing patent protection expected to last into the mid-2030s.

Iron deficiency remains a significant health issue among young people, affecting an estimated 2.4 million children in the US, according to Shield Therapeutics’ approval announcement. Adolescents are particularly vulnerable, with females at higher risk. Up to 40% of females aged 12 to 21 may experience iron deficiency due to factors such as menstruation, rapid growth during adolescence and increased iron requirements during puberty.

Some common symptoms of low iron in children include fatigue, paleness, irritability or difficulty keeping up in school or activities.

Untreated iron deficiency can lead to a range of complications in children and teenagers, including fatigue, developmental delays, impaired concentration and learning difficulties. Early diagnosis and treatment are therefore critical to prevent long-term health and cognitive effects.

Accrufer contains ferric maltol, a non-salt-based oral iron formulation designed to improve tolerability compared with traditional iron salts. The compound stabilizes iron with maltol, helping it remain intact until absorption in the small intestine, which may reduce gastrointestinal side effects that commonly limit adherence to conventional iron supplements.

Many oral iron therapies are associated with side effects such as nausea, constipation and stomach discomfort, which can make long-term treatment difficult for patients, especially children. Shield Therapeutics says Accrufer was developed to address these issues while effectively restoring iron levels.

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The FDA’s decision was supported by data from the FORTIS Phase III pediatric clinical trial, which evaluated Accrufer in adolescents with iron deficiency. In the study, pediatric patients aged 10 to 17 received twice-daily dosing of the therapy. After 12 weeks of treatment, patients experienced a clinically meaningful average hemoglobin increase of 1.1 g/dL, demonstrating the drug’s ability to improve iron levels.

The label expansion represents an important step for pediatric iron deficiency management, a condition that is both common and often undertreated. By providing a prescription oral therapy with demonstrated efficacy and tolerability, the approval could help address treatment adherence challenges in adolescents.

Looking ahead, Shield Therapeutics has indicated plans to pursue additional label expansions, including a potential pediatric suspension formulation that could enable treatment in younger children and infants as young as one month of age.