FibroGen Receives FDA Orphan Drug Designation for Roxadustat
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Dec 15 2025
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FibroGen 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."
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Analyst Views on FGEN
Wall Street analysts forecast FGEN stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for FGEN is 43.00 USD with a low forecast of 43.00 USD and a high forecast of 43.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
2 Analyst Rating
1 Buy
1 Hold
0 Sell
Moderate Buy
Current: 9.710
Low
43.00
Averages
43.00
High
43.00
Current: 9.710
Low
43.00
Averages
43.00
High
43.00
About FGEN
Kyntra Bio, Inc., formerly FibroGen, Inc., is a biopharmaceutical company. The Company is focused on development of novel therapies in oncology and rare disease. The Company continues to evaluate the development plan for the Phase III trial of roxadustat in anemia associated with lower-risk myelodysplastic syndrome (LR-MDS) in the U.S. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46, is in Phase II development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of FG-3180, an associated CD46-targeted PET biomarker. Roxadustat (EVRENZO) is approved in Europe, Japan, and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
FibroGen Receives FDA Orphan Drug Designation for Roxadustat in MDS Treatment
- Orphan Drug Designation: FibroGen's roxadustat has received FDA Orphan Drug Designation for treating approximately 58,000 MDS patients in the U.S., underscoring the urgent treatment gap and potentially providing the company with a competitive market advantage.
- Clinical Trial Progress: The company plans to submit the Phase 3 protocol for roxadustat in Q4 2025, aiming to improve treatment outcomes for patients with high transfusion burden, which is expected to significantly enhance patient quality of life.
- Market Potential: With about 50% of MDS patients reliant on transfusions and existing treatments achieving less than 50% efficacy, the oral administration of roxadustat is poised to meet the demand for convenient therapies, filling a critical market void.
- Strategic Collaboration: FibroGen is collaborating with Astellas to commercialize roxadustat across multiple markets including Japan and Europe, further strengthening its competitive position and influence in the global market.
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FibroGen Receives FDA Orphan Drug Designation for Roxadustat, Plans Phase 3 Submission in Q4 2025
- Orphan Drug Designation: FibroGen's roxadustat has received Orphan Drug Designation from the FDA for treating approximately 58,000 patients with lower-risk myelodysplastic syndromes (LR-MDS) in the U.S., highlighting a significant treatment gap and potentially enhancing the company's competitive position in this market.
- Clinical Trial Progress: The company plans to submit the Phase 3 protocol for roxadustat in Q4 2025, aiming to improve transfusion independence in patients with high transfusion burden, thereby solidifying its leadership in the MDS treatment space.
- Market Demand: Currently, about 50% of MDS patients require regular blood transfusions, with existing first-line treatments achieving less than 50% transfusion independence; roxadustat's clinical data indicates its potential to meet the urgent market need for new therapies.
- Strategic Collaboration: FibroGen is collaborating with Astellas to commercialize roxadustat across multiple markets, including Japan and Europe, which is expected to expand market share and enhance the product's global impact.
Continue Reading