- In April 2023, Sanofi announced promising results from its Phase 3 clinical trial evaluating sutimlimab-jome in patients with cold agglutinin disease (CAD), a rare autoimmune hemolytic anemia. The trial demonstrated significant reductions in hemolysis and transfusion needs, underscoring the potential of complement inhibition in addressing CAD symptoms. These findings reinforce Sanofi’s leadership in rare hematologic disorders and its ongoing investment in immunohematology innovations
- In March 2023, Apellis Pharmaceuticals received expanded regulatory approval in the European Union for Empaveli (pegcetacoplan) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare form of hemolytic anemia. The label extension follows strong clinical efficacy data showing durable hemoglobin stabilization and transfusion avoidance. This move strengthens Apellis’ global presence and highlights the increasing acceptance of complement-targeting therapies in rare anemia treatment
- In March 2023, Grifols S.A. introduced a new plasma-derived therapy under investigation for autoimmune hemolytic anemia, leveraging its expertise in immunoglobulin and plasma protein therapeutics. The company announced a new clinical trial site expansion across North America and Europe, aimed at accelerating development timelines. Grifols’ innovation in plasma therapies reflects the growing demand for targeted, well-tolerated treatment options for chronic hemolytic conditions
- In February 2023, Agios Pharmaceuticals, Inc. reported positive long-term data from its pivotal Phase 2 study of mitapivat in adults with pyruvate kinase deficiency (PKD), a hereditary hemolytic anemia. The data demonstrated sustained hemoglobin improvements and a favorable safety profile. Agios also revealed plans to explore mitapivat’s potential in thalassemia and sickle cell disease, marking a strategic expansion into broader hemolytic anemia indications and reinforcing its commitment to genetically driven hematologic disorders
- In January 2023, Regeneron Pharmaceuticals and Alnylam Pharmaceuticals expanded their RNA interference (RNAi) collaboration to include investigational programs targeting rare blood disorders, including autoimmune hemolytic anemia. The collaboration aims to harness Alnylam’s RNAi platform to silence disease-driving genes, while leveraging Regeneron’s expertise in antibody therapies. This joint initiative highlights a trend toward precision medicine approaches in treating rare hemolytic anemias, focusing on sustainable, long-term disease control
