Efficacy

Accrufer (Ferric Maltol) for Iron Deficiency

Accrufer, known generically as ferric maltol, is a medication approved for the treatment of iron deficiency in adults. Iron deficiency is a common condition characterized by a decrease in the number of red blood cells or hemoglobin in the blood due to a lack of iron. This can lead to symptoms such as fatigue, weakness, and shortness of breath. Accrufer works by providing a source of iron that is readily absorbed by the gastrointestinal tract, thereby increasing the iron levels in the body and helping to alleviate the symptoms of iron deficiency.

Clinical trials have demonstrated the efficacy of Accrufer in improving iron levels. In a pivotal phase 3 study, patients with iron deficiency anemia who were intolerant to or had an inadequate response to previous iron supplementation therapy showed significant increases in hemoglobin levels after 12 weeks of treatment with ferric maltol. The results indicated that Accrufer was effective in correcting iron deficiency and was well-tolerated by patients.

Reblozyl (Luspatercept) for Iron Deficiency

Reblozyl, with the generic name luspatercept, is another medication that has been studied for its efficacy in treating anemia, which can be caused by iron deficiency. Luspatercept is a recombinant fusion protein that is designed to regulate late-stage erythropoiesis (red blood cell production). It is primarily approved for the treatment of anemia in adults with beta thalassemia and myelodysplastic syndromes, conditions that are associated with ineffective erythropoiesis and consequently may lead to iron overload due to frequent blood transfusions.

While Reblozyl is not specifically approved for the treatment of iron deficiency anemia without underlying conditions like beta thalassemia or myelodysplastic syndromes, its mechanism of action suggests potential benefits in improving anemia by enhancing erythropoiesis. Clinical trials have shown that Reblozyl can significantly reduce the transfusion burden in patients with beta thalassemia and lower-risk myelodysplastic syndromes. However, its direct efficacy in treating iron deficiency without these conditions has not been established, and its use for this purpose would be considered off-label.