FibroGen Inc. (NASDAQ:FGEN) saw its stock jump nearly 10% after receiving encouraging feedback from the U.S. Food and Drug Administration (FDA) regarding the development of roxadustat for treating anemia in patients with low-risk myelodysplastic syndromes (LR-MDS) who rely heavily on red blood cell transfusions.
The FDA’s positive response followed a Type C meeting where FibroGen presented a post-hoc analysis from its Phase 3 MATTERHORN trial. The data revealed that 36% of patients with high transfusion needs who were treated with roxadustat achieved at least 56 days of transfusion independence—compared to just 7% of patients receiving a placebo.
“We are very pleased with the FDA’s feedback regarding roxadustat in patients with LR-MDS and anemia with high transfusion requirements. This indication, despite recent approvals, still represents a patient population with significant unmet need,” said Thane Wettig, CEO of FibroGen.
Looking ahead, the company intends to launch a new Phase 3 trial enrolling around 200 LR-MDS patients who require at least four units of packed red blood cells across two consecutive 8-week periods. Eligible participants must also be either refractory, intolerant, or ineligible for erythropoiesis-stimulating agent (ESA) therapy.
FibroGen emphasized that roxadustat’s oral formulation could present a more convenient alternative to injectable treatments currently on the market for this condition. The full clinical protocol for the upcoming study is expected to be submitted to the FDA in the fourth quarter of 2025.
Commenting on the trial plans, Dr. Amer Zeidan of Yale School of Medicine—who will lead the study—stated: “There is a significant unmet need for new, effective oral agents for this patient population,” despite recent advances involving injectables.
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