FibroGen granted orphan drug designation for roxadustat in MDS

FibroGen (FGEN) announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes. “The Orphan Drug Designation granted to roxadustat for MDS underscores the significant treatment gap in this indication, and highlights patients’ need for additional convenient treatments that can provide durable response,” said Thane Wettig, Chief Executive Officer of FibroGen. “Roxadustat showed an improvement in transfusion-independence in a subset of patients with high transfusion burden in a post-hoc analysis from the Phase 3 MATTHERHORN trial, which along with its favorable tolerability profile and oral route of administration has the ability to set it apart from current second-line treatments. Our team is finalizing the Phase 3 protocol in this patient population for submission to the FDA in the fourth quarter of 2025.”