Roxadustat Granted Orphan Drug Designation for the Treatment of Myelodysplastic Syndromes by the U.S. Food and Drug Administration

Rhea-AI Summary

FibroGen (NASDAQ: FGEN) announced that the U.S. FDA granted Orphan Drug Designation to roxadustat for the treatment of myelodysplastic syndromes (MDS) on Dec 15, 2025. The designation may provide fee exemptions, clinical development incentives, and 7 years of U.S. market exclusivity if approved. A post-hoc analysis from the Phase 3 MATTERHORN trial reported roxadustat benefit for transfusion independence in a subset of patients with high transfusion burden. FibroGen is finalizing a Phase 3 protocol for this MDS population and plans to submit it to the FDA in Q4 2025. The company noted ~58,000 LR-MDS patients in the U.S., with ~85% experiencing anemia and many facing transfusion dependence and related complications.

Positive

• FDA Orphan Drug designation for roxadustat (Dec 15, 2025)
• Post-hoc MATTERHORN analysis showed transfusion-independence benefit in high-burden subset
• Phase 3 protocol targeted for submission in Q4 2025

Negative

• Efficacy evidence cited is from a post-hoc subset analysis, not a primary endpoint
• Phase 3 protocol is being finalized and not yet submitted

Key Figures

LR-MDS patients US 58,000 patients Lower-risk myelodysplastic syndromes population in the U.S.

Anemia prevalence 85% Share of LR-MDS patients in U.S. suffering from anemia

First-line transfusion independence Less than 50% Proportion of patients achieving transfusion independence on current first-line treatments

Orphan prevalence threshold Fewer than 200,000 people U.S. definition of rare disease for Orphan Drug Designation

Market exclusivity 7 years U.S. market exclusivity granted after approval under Orphan Drug Designation

Market Reality Check

$8.38 Last Close

Volume Pre-news volume of 11,658 shares is below the 20-day average of 26,240 (relative volume 0.44). low

Technical Shares at $8.38 were trading below the 200-day MA of $8.89, and about 61.8% under the 52-week high of $21.94.

Peers on Argus

Before this FDA orphan designation, FGEN was down 3.68% with low volume, while close biotech peers showed a mixed picture: VTVT up 4.15%, PDSB down 4.5%, RLMD down 3.79%, and SER down 3.7%. No coordinated sector move was evident.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 02	Conference appearance	Neutral	+0.5%	Participation in rare disease investor summit with panel and 1x1 meetings.
Nov 10	Earnings and update	Positive	-0.3%	Q3 2025 results, China sale proceeds, debt repayment, and pipeline progress.
Nov 03	Earnings date notice	Neutral	-0.1%	Scheduled Q3 2025 earnings call and webcast announcement.
Sep 24	Clinical trial start	Positive	+3.4%	Initiation of Phase 2 monotherapy trial of FG-3246 in mCRPC.
Sep 02	Asset sale	Positive	+3.0%	Completion of FibroGen China sale and extension of cash runway into 2028.

Pattern Detected

Recent value-creating corporate and clinical updates have more often seen aligned, modestly positive price reactions, with only one notable divergence on earnings.

Recent Company History

Over the past six months, FibroGen has executed several key steps. On Sep 2, it closed the sale of FibroGen China for about $220M, repaid debt, and extended cash runway into 2028, with shares rising 2.96%. A Phase 2 FG-3246 trial launch on Sep 24 drove a 3.43% gain. Q3 results on Nov 10 highlighted the China sale and balance sheet improvement but saw a slight -0.27% reaction. A rare-disease conference appearance and other updates had small, generally aligned moves, framing today’s orphan designation as another step in the roxadustat/MDS strategy.

Market Pulse Summary

The stock is up +5.0% following this news. A strong positive reaction aligns with FibroGen’s history of meaningful moves on clinical milestones, as seen with prior FG‑3246 updates averaging 3.88%. The orphan status for roxadustat in lower‑risk MDS, plus plans to file a Phase 3 protocol in 4Q 2025, added regulatory and development clarity. Investors would still need to weigh trial execution risk and future financing needs, given the company’s small $35.2M market cap and prior reverse split.

Key Terms

orphan drug designation regulatory

"has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

phase 3 medical

"on track to file the Phase 3 protocol in the fourth quarter of 2025"

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

post-hoc analysis medical

"in a post-hoc analysis from the Phase 3 MATTHERHORN trial, which along with its"

Post-hoc analysis is an examination of data carried out after an experiment, trial, or reporting period to look for patterns or explanations that were not specified beforehand. It matters to investors because such findings can suggest new opportunities or risks but are more likely to be chance results than preplanned conclusions, so they require independent confirmation before being treated as reliable — like noticing a pattern on a map after a trip and then testing it on the next journey.

AI-generated analysis. Not financial advice.

• Company is on track to file the Phase 3 protocol in the fourth quarter of 2025
  

SAN FRANCISCO, Dec. 15, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS).

“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.”

There are approximately 58,000 patients diagnosed with LR-MDS in the U.S. with 85% of them suffering from anemia. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusions. Transfusion-dependent patients suffer higher rates of complications and decreased quality of life. Current first-line treatments lead to transfusion independence in less than 50% of patients and relief is often temporary with limited options for second line and beyond treatments. In a post-hoc analysis from the Phase 3 MATTERHORN trial, roxadustat demonstrated transfusion independence benefits compared to placebo in patients with high transfusion burden.

The FDA Orphan Drug Designation is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Benefits of the designation may include exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. following drug approval.

About Myelodysplastic Syndromes Anemia
Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S, of which 77% are 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. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion-dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease. Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin.

Roxadustat is approved in Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa.

About FibroGen  
FibroGen, Inc. is a biopharmaceutical company focused on development of novel therapies at the frontiers of cancer biology and anemia. Roxadustat (爱瑞卓^®, EVRENZO^TM) is currently 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. The Company continues to evaluate the development plan for the Phase 3 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 2 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. For more information, please visit www.fibrogen.com. 

Forward-Looking Statements 
This release contains forward-looking statements regarding FibroGen’s strategy, future plans and prospects, including statements regarding its commercial products and clinical programs and those of it and its collaboration partners Fortis and UCSF. These forward-looking statements include, but are not limited to, statements regarding the efficacy, safety, convenience, and potential clinical or commercial success of FibroGen products and product candidates, statements about regulatory interactions, and statements about FibroGen’s plans and objectives. These forward-looking statements are typically identified by use of terms such as “may,” “will”, “should,” “on track,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue” and similar words, although some forward-looking statements are expressed differently. FibroGen’s actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of its various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in FibroGen’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, each as filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and FibroGen undertakes no obligation to update any forward-looking statement in this press release, except as required by law. 

For Investor Inquiries:

David DeLucia, CFA
Senior Vice President and Chief Financial Officer
ir@fibrogen.com

FAQ

What does the FDA Orphan Drug Designation mean for FibroGen (FGEN) and roxadustat?

The designation may provide fee exemptions, clinical development incentives, and 7 years of U.S. market exclusivity after approval.

When will FibroGen (FGEN) submit the Phase 3 protocol for roxadustat in MDS?

FibroGen is finalizing the Phase 3 protocol and plans to submit it to the FDA in Q4 2025.

What clinical evidence supports roxadustat for MDS in the announcement for FGEN?

A post-hoc analysis from the Phase 3 MATTERHORN trial showed transfusion-independence benefits in a subset of patients with high transfusion burden.

How large is the target U.S. patient population mentioned for roxadustat (FGEN)?

The announcement cites approximately 58,000 lower-risk MDS patients in the U.S., with about 85% experiencing anemia.

Does the Orphan Drug Designation mean roxadustat is approved for MDS?

No; the designation is a regulatory status that can grant incentives but is not an approval for treatment use.