The Food and Drug Administration (FDA) has approved Accrufer (ferric maltol; Shield Therapeutics) for the treatment of iron deficiency in adults.
Accrufer is an oral treatment that provides an alternative to salt-based oral iron therapies. According to the Company, with Accrufer, iron is absorbed from the ferric maltol molecule and generally does not cause gastrointestinal adverse events (ie, nausea, bloating, constipation) typically observed with salt-based therapies.
The approval was based on data from 3 placebo controlled trials: AEGIS 1 (inflammatory bowel disease [IBD] patients), AEGIS 2 (IBD patients), and AEGIS 3 (chronic kidney disease [CKD] patients). In AEGIS 1 and 2, the safety and efficacy of Accrufer was evaluated in 128 patients with quiescent IBD and baseline hemoglobin (Hb) concentrations between 9.5g/dL and 12g/dL (females) or 13g/dL (males) and ferritin <30µg/L. The primary efficacy outcome was the mean difference in Hb concentration from baseline to Week 12.
Results showed the least square (LS) mean difference from baseline Hb between Accrufer and placebo was 2.18g/dL (P <.0001) at Week 12. Following the placebo-controlled phase, IBD patients were transitioned to open label treatment for an additional 52 weeks. During the open label phase, the mean change in Hb concentration from baseline to Week 64 was 3.1g/dL.
In AEGIS 3, 167 patients with non-dialysis dependent CKD and baseline Hb concentrations between 8g/dL and 11g/dL were randomized 2:1 to receive Accrufer or placebo for 16 weeks. The major efficacy outcome was the mean difference in Hb concentration from baseline to Week 16. Findings from the study showed that the LS mean difference from baseline Hb between Accrufer and placebo was 0.52g/dL (P=.0149).
“The broad label that the FDA has granted provides a very strong signal as to the tolerability and efficacy profile of Accrufer and provides a novel and convenient treatment alternative to the millions of US patients who routinely suffer with iron deficiency,” said Jackie Mitchell, VP Regulatory Affairs of Shield Therapeutics. “We believe that this broad approval, together with the recent clinical trial data on Feraccru [name outside of US] that showed it to be noninferior in treatment effect to Ferinject/ Injectafer, the leading IV iron therapy, can lead to a change in the current paradigm for the treatment iron deficiency anemia.”
Accrufer is supplied in a capsule formulation containing 30mg of iron, as ferric maltol.
For more information visit shieldtherapeutics.com.