Shares of FibroGen, Inc. (NASDAQ:FGEN) climbed about 3% on Monday after the company said its drug candidate roxadustat has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of myelodysplastic syndromes (MDS).
The designation was awarded by the FDA’s Office of Orphan Products Development and comes with several potential benefits, including eligibility for certain fee waivers, development incentives and up to seven years of market exclusivity in the United States if the drug is approved. Orphan Drug status is reserved for therapies targeting rare conditions that affect fewer than 200,000 people nationwide.
“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.
FibroGen estimates that around 58,000 patients in the U.S. are diagnosed with low-risk MDS, with anemia affecting roughly 85% of this population. The company noted that existing first-line therapies achieve transfusion independence in fewer than half of patients, while treatment options beyond the first line remain limited.
Data from a post-hoc analysis of the Phase 3 MATTERHORN trial showed that roxadustat improved transfusion independence compared with placebo in patients with a high transfusion burden. FibroGen said it is in the process of finalizing its Phase 3 study protocol and plans to submit it to the FDA in the fourth quarter of 2025.
The company added that roxadustat’s oral formulation could help distinguish it from currently available second-line treatment options for patients with MDS.