FDA approves ferric maltol for iron deficiency in patients 10 years and up

The FDA has approved an expanded indication for ferric maltol (ACCRUFeR; Shield Therapeutics), extending use of the oral iron therapy to include pediatric patients aged 10 years and older with iron deficiency, following a priority review.¹

The decision broadens access beyond adults, for whom the therapy was first approved in 2019, and reflects growing clinical evidence supporting the safety and efficacy of ferric maltol in younger populations with iron deficiency and iron deficiency anemia.

Shield Therapeutics announced that the indication expansion allows ferric maltol to be used for the treatment of iron deficiency in both adult and pediatric patients aged 10 years and older. The approval was supported by results from a Phase 3 pediatric clinical trial confirming efficacy, safety, and tolerability of an oral liquid pediatric formulation in children with iron deficiency presenting as iron deficiency anemia.¹ The company also stated that it plans to seek a further indication of expansion to include children as young as 1 month of age, alongside submission of a new drug application for a pediatric suspension formulation used in that study.

Iron deficiency is among the most common nutritional and hematologic conditions worldwide and can affect patients across a wide range of ages and clinical settings. In pediatric patients, untreated iron deficiency and iron deficiency anemia may contribute to fatigue, impaired cognitive development, and reduced physical endurance. Oral iron therapies remain a cornerstone of treatment, but tolerability and adherence challenges have limited their effectiveness in some patients.

Adolescent data for ferric maltol

The FDA’s approval for pediatric patients aged 10 to 17 years was supported by efficacy data from the FORTIS clinical trial, which evaluated ferric maltol in adolescents with iron deficiency.² In that study, 24 pediatric patients received age-based dosing of ferric maltol capsules twice daily. Treatment resulted in a clinically meaningful average increase in hemoglobin of 1.1 g/dL at Week 12, an improvement the agency noted is comparable, on average, to the hemoglobin increase typically seen after a single blood transfusion.

Additional pediatric evidence cited by Shield Therapeutics came from the broader Phase 3 FORTIS/ST10-01-305 program, which included children from 1 month to 17 years of age with mild to moderate iron deficiency anemia.¹ In that open-label, randomized study, children aged 2 to 17 years were randomized to receive either ferric maltol or ferrous sulphate, while infants younger than 2 years all received ferric maltol. According to the company, the trial confirmed efficacy and tolerability and represents the final study in a comprehensive pediatric development program committed to both US and European regulators.

Ferric maltol is a non–non-salt-based oral iron therapy designed to improve gastrointestinal tolerability compared with traditional iron salts. Shield Therapeutics has described the drug as having a novel mechanism of absorption, which may allow for more predictable restoration of iron levels while minimizing gastrointestinal adverse effects associated with conventional oral iron products.¹ The therapy has been positioned as an option for patients who require oral iron but have difficulty tolerating existing formulations.

The expanded pediatric approval addresses a substantial unmet need, particularly for adolescents who may experience iron deficiency related to growth, dietary factors, or blood loss. Shield Therapeutics estimates that iron deficiency and iron deficiency anemia affect approximately 20 million people in the United States, representing a significant clinical and economic burden.

Safety of ferric maltol

According to the FDA, ferric maltol should not be used in patients with known hypersensitivity to the product or its excipients, those with hemochromatosis or other iron overload syndromes, or patients receiving repeated blood transfusions. The agency also advises avoiding ferric maltol during inflammatory bowel disease flares and highlights the risk of iron overload. The most common adverse reactions reported include flatulence, diarrhea, constipation, discolored feces, abdominal pain, nausea, vomiting, and abdominal discomfort or distension.²

With the expanded approval, ferric maltol becomes an FDA-approved oral iron option for adolescents with iron deficiency, while additional regulatory submissions may further broaden access to younger pediatric populations in the future.

Specifically, "Shield plans to file for a further extension of the indication to include children 1 month and above in conjunction with the submission of an NDA for a new pediatric formulation (ferric maltol suspension), which was used in the successful FORTIS Phase 3 study in this population. If approved, this formulation may also offer an alternative approach for adults who can’t swallow our current capsule formulation," Shield stated in the announcement.¹

References:

1. US FDA approves extension of the indication for ACCRUFeR® to include children 10 years and older with iron deficiency (ID). Shield Therapeutics. Press release. December 22, 2025. Accessed December 22, 2025. https://www.shieldtherapeutics.com/application/files/8817/6639/2917/STX_RNS_FDA_Approval_22Dec2025.pdf
2. FDA approves first prescription oral medicine for iron deficiency in pediatric patients ages 10 and older. FDA. Press release. December 22, 2025. Accessed December 22, 2025. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-prescription-oral-medicine-iron-deficiency-pediatric-patients-ages-10-and-older