Shield Therapeutics Reports Positive Phase 3 Pediatric Trial Outcomes for ACCRUFeR® Ferric Maltol in Pediatric Drugs Journal

FORTIS Trial Design and Pediatric Population

The FORTIS study (ST10-01-305) was an open-label, randomized Phase 3 trial assessing the safety, efficacy, tolerability, and palatability of ferric maltol versus ferrous sulfate in pediatric patients with iron deficiency anaemia (IDA). Participants ranged from 1 month to 17 years old, with mild to moderate IDA defined by serum ferritin <30 µg/L or ferritin <50 µg/L plus transferrin saturation <20%.

Two treatment cohorts were established: children aged 2 to 17 years were randomized 1:1, with 31 receiving ferric maltol and 30 ferrous sulfate; a younger group aged 1 month to under 2 years (N=4) all received ferric maltol. This trial completed Shield’s pediatric development programme commitments to the EMA and FDA.

Significant Haemoglobin Improvements Across Treatments

The FORTIS trial met its primary efficacy endpoint, demonstrating statistically and clinically significant haemoglobin increases from baseline in all groups (P<0.0001). Both ferric maltol and ferrous sulfate effectively improved iron metabolism over 12 weeks. The consistent haemoglobin gains across diverse pediatric ages indicate ferric maltol as a viable treatment for iron deficiency anaemia in very young children.

Additionally, ferric maltol significantly enhanced iron storage markers and offered improved tolerability and palatability compared to ferrous sulfate. This combination of effective iron repletion and better gastrointestinal tolerability is clinically important for pediatric patients who often experience side effects from traditional iron supplements.

Enhanced Safety and Tolerability Versus Ferrous Sulfate

The trial highlighted ferric maltol’s superior tolerability, with treatment-related adverse events in 6.5% of patients versus 13% for ferrous sulfate, representing a twofold reduction. No serious treatment-related adverse events occurred in either group, confirming safety in the pediatric population.

No patients discontinued ferric maltol due to adverse events, compared to one discontinuation in the ferrous sulfate arm. This improved tolerability may stem from ferric maltol’s unique absorption mechanism and stable, non-salt formulation, reducing gastrointestinal side effects common with traditional iron salts.

Broadened Age Indication Supported by Pediatric Data

As of the 28 July 2026 announcement, the 30 mg ferric maltol capsule is approved for adolescents and adults aged 10+ in the US under ACCRUFeR®, and for patients 12+ in the EU as FeRACCRU®. The FORTIS trial included children as young as 1 month, demonstrating safety and efficacy across a wider pediatric age range than current approvals.

Following these results, Shield plans to register a liquid ferric maltol formulation for children aged 2 years and older. This formulation aims to address an unmet need for younger pediatric patients unable to swallow capsules, expanding ACCRUFeR®/FeRACCRU®’s market reach and providing an effective oral iron therapy for infants and toddlers.

Peer-Reviewed Publication Validates Clinical Programme

The FORTIS trial findings were published in Pediatric Drugs, authored by Gass, D., Freiberg, A., Auth, M.K.H., and Russell, R.K., with Professor Richard Russell as Principal Investigator and corresponding author (DOI: 10.1007/s40272-026-00753-6). This peer-reviewed article underwent rigorous scientific scrutiny, enhancing the credibility of Shield’s clinical data.

Professor Russell noted that confirming ferric maltol’s safety and efficacy in children as young as 2 months is "an important milestone in expanding therapeutic options for pediatric patients," highlighting the need for safe, effective oral iron treatments.

ACCRUFeR® Market Leadership and Competitive Edge

Shield Therapeutics positions ACCRUFeR® as the leading branded prescription oral iron based on IQVIA Xponent PlanTrak data. It is the first and only FDA-approved oral iron therapy for iron deficiency with and without anaemia, reflecting strong commercial traction in the US market.

Iron deficiency and anaemia affect roughly 20 million people in the US, representing a $2.3 billion market. Ferric maltol’s novel absorption and improved tolerability demonstrated in FORTIS position ACCRUFeR® to capture market share from traditional iron salts. The pediatric indication expansion could unlock a significant underserved segment.

Global Commercial Partnerships and Licensing

Shield employs a partnership-driven strategy to maximize ACCRUFeR®/FeRACCRU®’s global reach. In the US, Shield has an exclusive multi-year collaboration with Viatris to commercialize ACCRUFeR®, including pediatric patients aged 10+. Outside the US, licenses exist with five specialty pharmaceutical companies.

In Europe and the UK, FeRACCRU® is marketed by Norgine B.V., which also covers Australia and New Zealand. Kye Pharmaceuticals Inc. commercializes the product in Canada. For Asia, Beijing Aosaikang Pharmaceutical Co., Ltd. holds rights for China, Hong Kong, Macau, and Taiwan; Korea Pharma Co., Ltd. for South Korea; and Medleap Pharma Company Limited (a VITAL-NET Inc. subsidiary) for Japan. This broad portfolio leverages local expertise and regulatory knowledge worldwide.

Patent Protection Secures Market Exclusivity Through Mid-2030s

ACCRUFeR®/FeRACCRU® is protected by patents extending to the mid-2030s, providing Shield and partners market exclusivity against generic competition during this period. This protection supports recouping R&D investments and maintaining premium pricing.

Potential pediatric exclusivity extensions may further prolong patent protection. Shield’s partnerships may also offer ongoing royalty revenues post-patent expiry, though commercial terms remain undisclosed.

Completion of Pediatric Development Programme

Shield fulfilled regulatory commitments to the EMA and FDA by completing the FORTIS trial, the final study in its pediatric development programme. This achievement removes regulatory barriers and supports pediatric label expansions based on robust clinical evidence.

The positive FORTIS results and peer-reviewed publication strengthen Shield’s regulatory submissions for the planned liquid formulation targeting children aged 2 years and older. Approval of this formulation would represent a major value inflection by addressing a large pediatric market currently lacking effective oral iron therapies. Regulatory timelines and approval likelihood remain subject to ongoing agency interactions.