Dec. 15 at 1:30 PM
FibroGen said its drug roxadustat received FDA Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS), granting development incentives such as potential fee waivers, financial support, and seven years of U.S. market exclusivity upon approval. The designation highlights a significant unmet need, as current therapies leave many low-risk MDS patients transfusion-dependent.
The company noted that most low-risk MDS patients in the U.S. suffer from anemia, and fewer than half achieve transfusion independence with first-line treatments. In a post-hoc analysis of the Phase 3 MATTERHORN trial, roxadustat showed improved transfusion independence versus placebo in patients with high transfusion burden. FibroGen plans to finalize its Phase 3 protocol for FDA submission in the fourth quarter of 2025, with oral administration potentially differentiating the drug from existing second-line options.
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