FibroGen Announces Positive Type C Meeting with the FDA for Roxadustat in Patients with Anemia Associated with Lower-Risk Myelodysplastic Syndromes
FibroGen (NASDAQ: FGEN) has received positive feedback from the FDA during a Type C meeting regarding roxadustat for treating anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high red blood cell transfusion burden.
The feedback was based on promising post-hoc analysis data from the MATTERHORN Phase 3 trial, where roxadustat showed significant efficacy: 36% (8/22) of patients achieved transfusion independence versus 7% (1/15) in the placebo group. The company plans to initiate a new Phase 3 trial with approximately 200 patients, with the protocol submission expected in Q4 2025.
The planned study will be randomized, double-blind, and placebo-controlled, targeting patients requiring ¡Ý4 pRBC units in consecutive 8-week periods who are refractory to, intolerant to, or ineligible for prior ESA therapy.
FibroGen (NASDAQ: FGEN) ha ricevuto un riscontro positivo dalla FDA durante un incontro di Tipo C riguardante roxadustat per il trattamento dell'anemia in pazienti con sindromi mielodisplastiche a basso rischio (LR-MDS) e un elevato fabbisogno di trasfusioni di globuli rossi.
Il feedback si basa su promettenti dati di analisi post-hoc dello studio di Fase 3 MATTERHORN, in cui roxadustat ha mostrato un'efficacia significativa: il 36% (8/22) dei pazienti ha raggiunto l'indipendenza dalle trasfusioni rispetto al 7% (1/15) nel gruppo placebo. L'azienda prevede di avviare un nuovo studio di Fase 3 con circa 200 pazienti, con la presentazione del protocollo prevista per il quarto trimestre del 2025.
Lo studio programmato sar¨¤ randomizzato, in doppio cieco e controllato con placebo, rivolto a pazienti che necessitano di ¡Ý4 unit¨¤ di globuli rossi concentrati in periodi consecutivi di 8 settimane e che sono refrattari, intolleranti o non idonei alla precedente terapia con ESA.
FibroGen (NASDAQ: FGEN) ha recibido comentarios positivos de la FDA durante una reuni¨®n de Tipo C sobre roxadustat para el tratamiento de la anemia en pacientes con s¨ªndromes mielodispl¨¢sicos de bajo riesgo (LR-MDS) y una alta carga de transfusiones de gl¨®bulos rojos.
Los comentarios se basaron en datos prometedores de an¨¢lisis post-hoc del ensayo de Fase 3 MATTERHORN, donde roxadustat mostr¨® una eficacia significativa: el 36% (8/22) de los pacientes lograron independencia de transfusiones frente al 7% (1/15) en el grupo placebo. La compa?¨ªa planea iniciar un nuevo ensayo de Fase 3 con aproximadamente 200 pacientes, con la presentaci¨®n del protocolo prevista para el cuarto trimestre de 2025.
El estudio planificado ser¨¢ aleatorizado, doble ciego y controlado con placebo, dirigido a pacientes que requieren ¡Ý4 unidades de gl¨®bulos rojos en periodos consecutivos de 8 semanas y que son refractarios, intolerantes o no elegibles para la terapia previa con ESA.
FibroGen (NASDAQ: FGEN)? ??? ?????????(LR-MDS) ?? ? ??? ?? ??? ?? ?? ??? ?? ??????? ?? FDA?? Type C ???? ???? ???? ?????.
? ???? MATTERHORN 3? ??? ?? ?? ???? ??? ???, ??????? ???? ??? ?????: ??? 36%(8/22)? ?? ???? ?????, ???? 7%(1/15)? ??????. ??? ? 200?? ??? ???? ?? ??? 3? ??? ??? ????, ???? ??? 2025? 4??? ???? ????.
??? ??? ?????, ?????, ?? ?? ????, ?? 8? ?? ?? ¡Ý4??? ??? ??? ???? ?? ESA ??? ??? ??? ???? ??? ???? ???.
FibroGen (NASDAQ : FGEN) a re?u un retour positif de la FDA lors d'une r¨¦union de type C concernant le roxadustat pour le traitement de l'an¨¦mie chez les patients atteints de syndromes my¨¦lodysplasiques ¨¤ faible risque (LR-MDS) et pr¨¦sentant une forte charge transfusionnelle en globules rouges.
Ce retour s'appuie sur des donn¨¦es prometteuses d'analyses post-hoc de l'essai de phase 3 MATTERHORN, o¨´ le roxadustat a montr¨¦ une efficacit¨¦ significative : 36 % (8/22) des patients ont atteint l'ind¨¦pendance transfusionnelle contre 7 % (1/15) dans le groupe placebo. L'entreprise pr¨¦voit de lancer un nouvel essai de phase 3 avec environ 200 patients, avec une soumission du protocole pr¨¦vue au quatri¨¨me trimestre 2025.
L'¨¦tude pr¨¦vue sera randomis¨¦e, en double aveugle et contr?l¨¦e par placebo, ciblant les patients n¨¦cessitant ¡Ý4 unit¨¦s de concentr¨¦s de globules rouges sur des p¨¦riodes cons¨¦cutives de 8 semaines, qui sont r¨¦fractaires, intol¨¦rants ou non ¨¦ligibles ¨¤ une th¨¦rapie ESA ant¨¦rieure.
FibroGen (NASDAQ: FGEN) hat w?hrend eines Type-C-Meetings positive R¨¹ckmeldungen von der FDA bez¨¹glich Roxadustat zur Behandlung von An?mie bei Patienten mit niedrigrisiko Myelodysplastischem Syndrom (LR-MDS) und hoher Transfusionslast erhalten.
Das Feedback basierte auf vielversprechenden post-hoc Analysedaten aus der MATTERHORN Phase-3-Studie, in der Roxadustat eine signifikante Wirksamkeit zeigte: 36 % (8/22) der Patienten erreichten Transfusionsunabh?ngigkeit gegen¨¹ber 7 % (1/15) in der Placebo-Gruppe. Das Unternehmen plant den Start einer neuen Phase-3-Studie mit etwa 200 Patienten, wobei die Protokolleinreichung f¨¹r das vierte Quartal 2025 erwartet wird.
Die geplante Studie wird randomisiert, doppelblind und placebokontrolliert sein und sich an Patienten richten, die ¡Ý4 Einheiten gepackter roter Blutk?rperchen in aufeinanderfolgenden 8-Wochen-Zeitr?umen ben?tigen und die gegen¨¹ber vorheriger ESA-Therapie refrakt?r, intolerant oder nicht geeignet sind.
- Post-hoc analysis showed 36% transfusion independence rate vs 7% for placebo
- FDA agreement on key Phase 3 trial design elements
- Potential advantage as an oral treatment option in a market dominated by injectables
- Clear pathway established for regulatory advancement
- Additional Phase 3 trial required before potential approval
- Company still evaluating internal development vs partnership options
- Timeline to market extended due to new trial requirement
Insights
FDA's positive Type C meeting feedback advances FibroGen's roxadustat toward Phase 3 trials for LR-MDS anemia patients with high transfusion needs.
FibroGen's positive FDA Type C meeting represents a significant regulatory milestone for roxadustat in treating anemia associated with lower-risk myelodysplastic syndromes (LR-MDS). The FDA has agreed to key Phase 3 trial design elements, validating FibroGen's development strategy based on promising post-hoc analysis from their MATTERHORN study.
The post-hoc analysis revealed impressive efficacy in high transfusion burden patients, with
The regulatory pathway is now clearly defined with agreement on critical elements: patient population (¡Ý4 pRBC units in consecutive 8-week periods), dose regimen, and safety management protocols. This alignment significantly reduces regulatory uncertainty and streamlines the development process.
The oral administration route of roxadustat represents a potential competitive advantage in a market dominated by injectable therapies. Despite recent approvals in this space, the CEO correctly identifies substantial unmet needs, particularly for convenient oral treatment options.
From a regulatory perspective, this Type C meeting success doesn't guarantee approval but establishes a clear development framework with FDA buy-in. The planned 200-patient Phase 3 study with either 8-week or 16-week RBC transfusion independence as primary endpoint provides a well-defined path to potential market authorization for this specialized indication with significant unmet medical needs.
Roxadustat shows promising potential as first oral therapy for high-burden LR-MDS anemia patients, addressing critical treatment gap despite recent approvals.
The post-hoc analysis from MATTERHORN reveals roxadustat's substantial clinical benefit in a particularly challenging patient population. Achieving
The mechanism of action is particularly noteworthy. As a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), roxadustat works by mimicking the body's response to hypoxia, increasing endogenous erythropoietin production and improving iron utilization. This differs fundamentally from currently approved therapies like luspatercept (a TGF-beta ligand trap) and implies potential efficacy in different patient subsets.
The oral administration route addresses a crucial unmet need in LR-MDS treatment. Current approved therapies require subcutaneous or intravenous administration, creating substantial burden for this predominantly elderly patient population who often have limited mobility and require frequent healthcare visits. An effective oral option could dramatically improve treatment adherence and quality of life.
For context, transfusion dependence in LR-MDS patients is associated with significantly worse outcomes - including reduced survival, increased complications, iron overload issues, and frequent hospital visits. The 5-fold increase in transfusion independence versus placebo suggests roxadustat could meaningfully improve both clinical outcomes and quality of life metrics.
The involvement of Dr. Amer Zeidan, a recognized authority in myeloid malignancies, as principal investigator adds credibility to this development program. His emphasis on the continuing unmet need despite recent approvals aligns with the clinical reality that current treatments leave many patients without adequate options, particularly those seeking oral therapies with favorable safety profiles.
- FibroGen has reached agreement with the FDA on important design elements for a pivotal Phase 3 clinical trial for roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high red blood cell (RBC) transfusion burden
- Company intends to file the Phase 3 protocol in the fourth quarter of 2025
SAN FRANCISCO, Aug. 07, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc.?(NASDAQ: FGEN) today announced positive feedback from its Type C meeting with the FDA, supporting the advancement of roxadustat for the treatment of anemia in patients with LR-MDS and high RBC transfusion burden, based on a post-hoc subgroup analysis from the MATTERHORN Phase 3 trial.
¡°We are very pleased with the feedback we received from the FDA regarding roxadustat in patients with LR-MDS and anemia with high transfusion burden. This indication, despite recent approvals, still represents a patient population with significant unmet need,¡± said Thane Wettig, Chief Executive Officer of FibroGen. ¡°We believe roxadustat¡¯s differentiated mechanism of action, favorable tolerability profile, and oral route of administration can potentially be an important addition to the treatment options for patients with high transfusion burden. We are starting preparations for the Phase 3 trial, while evaluating internal development and potential partnership opportunities for this late-stage program. We plan to submit the Phase 3 trial protocol to the FDA in the fourth quarter of this year.¡±
¡°Anemia is a major cause of morbidity and complications in patients with LR-MDS, especially those with high transfusion burden, and is often associated with poor quality of life and shortened survival. While we have recent approvals of injectable drugs for this indication, there is a significant unmet need for novel, effective oral agents for this patient population,¡± added Amer Zeidan, M.B.B.S, M.H.S., Professor of Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center, and the global principal investigator of the planned Phase 3 study. ¡°Roxadustat has already shown promising efficacy in this group of patients in the post-hoc analysis of the MATTERHORN study, and I am glad we have agreed on a pathway with the regulators to explore the full potential of roxadustat in the upcoming Phase 3 trial. I am excited by the prospect of roxadustat potentially becoming a novel, safe, convenient, and effective therapy for LR-MDS patients with high transfusion burden.¡±
FibroGen requested the Type C meeting based on the findings of a post-hoc analysis of data from the Phase 3 MATTERHORN trial of roxadustat in anemia-associated with LR-MDS. In patients with high RBC transfusion burden at baseline (¡Ý4 units over 8 weeks1), a pronounced treatment effect was observed:
The planned Phase 3 trial will assess the safety and efficacy of roxadustat in a randomized, double-blind, placebo-controlled design in approximately 200 patients with LR-MDS. Alignment was reached with the FDA on the patient population (patients requiring ¡Ý 4 pRBC units in two consecutive 8-week periods prior to randomization, who are refractory to, intolerant to, or ineligible for prior erythropoiesis-stimulating agents (ESA) therapy), dose regimen, as well as management of potential thrombotic risk through eligibility and dose modification and discontinuation criteria. As the primary endpoint for the study, the Company is considering either 8-week or 16-week RBC TI.
FibroGen plans to submit the full Phase 3 protocol to the FDA in the fourth quarter of 2025.
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, thereof
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 in clinical development for chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted by the China Health Authority.
Roxadustat is approved in China, 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 accelerating the development of novel therapies at the frontiers of cancer biology. Roxadustat (°®Èð׿?, EVRENZO?) is currently approved in China, Europe, Japan, and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. Roxadustat is in clinical development for chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted for review by the China Health Authority. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46 is in development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of an associated CD46-targeted PET biomarker. In addition, FibroGen has expanded its research and development portfolio to include two immuno-oncology product candidates for the treatment of solid tumors. For more information, please visit .
Forward-Looking Statements
This release contains forward-looking statements regarding FibroGen¡¯s strategy, future plans and prospects, including statements regarding its clinical programs . 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 regarding study design, and patient and disease profile, 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.
Disclosure: Dr. Amer Zeidan has consulted and received honoraria from Fibrogen. The views expressed are his personal views and not necessarily those of his employer.
For Investor Inquiries:
David DeLucia, CFA
Senior Vice President and Chief Financial Officer
[email protected]
1Platzbecker U, et al. Blood. 2019;133(10):1020-1030.
FAQ
What were the results of FibroGen's Type C meeting with FDA for roxadustat?
What efficacy did roxadustat show in the MATTERHORN trial for MDS patients?
How many patients will be enrolled in FibroGen's new Phase 3 trial for roxadustat?
When will FibroGen submit the Phase 3 protocol for roxadustat in MDS?
What patient population will FibroGen's Phase 3 trial target?
