Shield Therapeutics plc announced that the US Food and Drug Administration has accepted ACCRUFeR/FeRACCRU (ferric maltol) as a Clinical Supplement and assigned Priority Review to extend the indication for treating iron deficiency anemia in adolescents aged 10 years and above. Pending successful review, approval is anticipated in 2026.
The regulatory milestone follows positive results from the Phase 3 pediatric clinical trial (FORTIS/ST10-01-305) that confirmed the efficacy, safety, and tolerability of the new oral liquid pediatric formulation in children with iron deficiency anemia. Shield submitted the clinical supplement application to the FDA in June 2025.
Phase 3 Trial Results Drive Regulatory Progress
The open-label randomized Phase 3 study included children aged 1 month to 17 years with mild to moderate iron deficiency anemia, who also had serum ferritin levels below 30 μg/L or ferritin levels below 50 μg/L and transferrin saturation below 20%. Children aged 2 to 17 years were randomized 1:1 to receive either ferric maltol (N=31) or ferrous sulphate (N=30). Children 1 month to under 2 years (N=4) were all assigned to receive ferric maltol treatment.
The FDA granted Priority Review for the supplement as it supports the extension of the label to a pediatric population based on a final agreed pediatric study report. The full data sets, including secondary endpoints and pharmacokinetic sub-study parameters, will be submitted for peer-review and subsequent presentation/publication.
Expanding Pediatric Access
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 the current capsule formulation.
Dr Jackie Mitchell, VP of Quality, RA and Clinical Development at Shield, commented: "We are delighted to be able to progress a further significant expansion of the patient population to adolescents, who can benefit from a much needed safe and effective oral iron treatment."
Global Regulatory Strategy
Shield's licensing partner in the EU, Norgine B.V., also filed a regulatory submission to the EMA in Q2 2025 for the approval of FeRACCRU (ferric maltol) in the adolescent population. Pending successful review, approval is also anticipated in 2026.
Market Opportunity and Unmet Need
Together, iron deficiency and iron deficiency with anemia affect about 20 million people in the US and represent a $2.3 billion market opportunity. As the first and only FDA approved oral iron to treat iron deficiency with or without anemia, ACCRUFeR has the potential to meet an important unmet medical need for both physicians and patients.
ACCRUFeR/FeRACCRU (ferric maltol) is a novel, stable, non-salt-based oral therapy for adults with iron deficiency with or without anemia. The drug has a novel mechanism of absorption compared to other oral iron therapies and has been shown to be an efficacious and well-tolerated therapy in a range of clinical trials.
Following the announcement, shares in Shield Therapeutics climbed 38% to 8.72 pence on Thursday morning in London, reflecting investor confidence in the regulatory progress and market expansion potential.
