FibroGen Receives Positive FDA Feedback for Roxadustat in Treating Anemia in LR-MDS Patients with High RBC Transfusion Burden.

FibroGen has received positive feedback from the FDA regarding roxadustat for treating anemia in patients with low-risk myelodysplastic syndromes (LR-MDS) and high red blood cell transfusion burden. The company plans to submit a Phase 3 trial protocol to the FDA in Q4 and is considering internal development and partnership opportunities for the late-stage program. Roxadustat has a differentiated mechanism of action and a favorable tolerability profile.

FibroGen, Inc. (NASDAQ: FGEN) has received positive feedback from the FDA regarding the use of roxadustat for treating anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high red blood cell transfusion burden. The company intends to file a Phase 3 trial protocol with the FDA in the fourth quarter of 2025, following a successful Type C meeting with the regulatory body.

The Type C meeting was requested based on a post-hoc analysis of data from the MATTERHORN Phase 3 trial, which demonstrated that roxadustat significantly improved transfusion independence in patients with high RBC transfusion burden. In the analysis, 36% of patients on roxadustat achieved transfusion independence for at least 56 days compared to 7% on placebo within 28 weeks (nominal p-value of 0.041) [1].

The planned Phase 3 trial will assess the safety and efficacy of roxadustat in approximately 200 patients with LR-MDS. The study will be a randomized, double-blind, placebo-controlled design and will evaluate patients requiring at least four packed red blood cell (pRBC) units in two consecutive 8-week periods prior to randomization, who are refractory to, intolerant to, or ineligible for prior erythropoiesis-stimulating agents (ESA) therapy [2].

FibroGen is currently preparing for the Phase 3 trial while evaluating internal development and potential partnership opportunities for this late-stage program. The company plans to submit the full Phase 3 protocol to the FDA in the fourth quarter of 2025.

Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis by increasing endogenous production of erythropoietin, improving iron absorption and mobilization, and downregulating hepcidin [2].

Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. The annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S., with 77% considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin 8 g/dL) at some point during the course of their disease [2].

References:
[1] https://www.nasdaq.com/press-release/fibrogen-announces-positive-type-c-meeting-fda-roxadustat-patients-anemia-associated
[2] https://www.biospace.com/press-releases/fibrogen-announces-positive-type-c-meeting-with-the-fda-for-roxadustat-in-patients-with-anemia-associated-with-lower-risk-myelodysplastic-syndromes