Geron Announces European Commission Approval of RYTELO® (imetelstat), a First-in-Class Telomerase Inhibitor, for the Treatment of Adults With Transfusion-Dependent Anemia Due to Lower-Risk MDS
Geron (GERN) has received European Commission (EC) approval for RYTELO® (imetelstat), the first-in-class telomerase inhibitor for treating adults with transfusion-dependent anemia due to lower-risk MDS (LR-MDS). The approval applies to patients without del-5q abnormality who are either ineligible for or had unsatisfactory response to erythropoietin-based therapy.
The approval is based on the IMerge Phase 3 clinical trial results, which showed significant reduction in red blood cell transfusion dependency during the first 24 weeks compared to placebo. The most common adverse reactions included neutropenia (69%) and thrombocytopenia (63%), typically resolving within four weeks.
RYTELO's marketing authorization covers all 27 EU member states, Iceland, Norway, and Liechtenstein. Geron plans to commercialize RYTELO in select EU countries starting in 2026, pending reimbursement approvals. The drug maintains orphan drug designation, providing 10-year market exclusivity, with patent exclusivity anticipated until 2038.
Geron (GERN) ha ricevuto l'approvazione della Commissione Europea (CE) per RYTELO® (imetelstat), il primo inibitore della telomerasi della sua classe per il trattamento degli adulti con anemia da trasfusione dipendente a causa di MDS a basso rischio (LR-MDS). L'approvazione si applica a pazienti senza anomalia del del-5q che sono o non idonei o hanno avuto una risposta insoddisfacente alla terapia a base di eritropoietina.
L'approvazione si basa sui risultati del trial clinico IMerge di Fase 3, che ha mostrato una significativa riduzione della dipendenza dalle trasfusioni di globuli rossi durante le prime 24 settimane rispetto al placebo. Le reazioni avverse più comuni includevano neutropenia (69%) e trombocitopenia (63%), che di solito si risolvono entro quattro settimane.
L'autorizzazione al commercio di RYTELO copre tutti i 27 stati membri dell'UE, Islanda, Norvegia e Liechtenstein. Geron prevede di commercializzare RYTELO in alcuni paesi dell'UE a partire dal 2026, in attesa delle approvazioni per il rimborso. Il farmaco mantiene la designazione di farmaco orfano, fornendo 10 anni di esclusività di mercato, con esclusività di brevetto prevista fino al 2038.
Geron (GERN) ha recibido la aprobación de la Comisión Europea (CE) para RYTELO® (imetelstat), el primer inhibidor de telomerasa de su clase para tratar a adultos con anemia dependiente de transfusiones debido a MDS de bajo riesgo (LR-MDS). La aprobación se aplica a pacientes sin la anomalía del del-5q que son inelegibles o han tenido una respuesta insatisfactoria a la terapia basada en eritropoietina.
La aprobación se basa en los resultados del ensayo clínico IMerge de Fase 3, que mostró una reducción significativa en la dependencia de transfusiones de glóbulos rojos durante las primeras 24 semanas en comparación con el placebo. Las reacciones adversas más comunes incluyeron neutropenia (69%) y trombocitopenia (63%), que generalmente se resuelven en un plazo de cuatro semanas.
La autorización de comercialización de RYTELO cubre los 27 estados miembros de la UE, Islandia, Noruega y Liechtenstein. Geron planea comercializar RYTELO en algunos países de la UE a partir de 2026, a la espera de aprobaciones de reembolso. El medicamento mantiene la designación de medicamento huérfano, proporcionando 10 años de exclusividad en el mercado, con exclusividad de patente anticipada hasta 2038.
Geron (GERN)은 RYTELO® (imetelstat)에 대해 유럽연합 집행위원회(EC)의 승인을 받았습니다. 이는 저위험 MDS(LR-MDS)로 인한 수혈 의존성 빈혈 치료를 위한 첫 번째 클래스의 텔로머라제 억제제입니다. 이 승인은 del-5q 이상이 없는 환자에게 적용되며, 이들은 에리트로포이에틴 기반 치료에 대해 부적격이거나 불만족스러운 반응을 보였습니다.
승인은 IMerge 3상 임상 시험 결과를 기반으로 하며, 이는 위약에 비해 첫 24주 동안 적혈구 수혈 의존도가 유의미하게 감소했음을 보여주었습니다. 가장 흔한 부작용으로는 호중구감소증(69%)과 혈소판감소증(63%)이 있으며, 일반적으로 4주 이내에 해결됩니다.
RYTELO의 마케팅 승인은 27개 EU 회원국, 아이슬란드, 노르웨이 및 리히텐슈타인을 포함합니다. Geron은 2026년부터 일부 EU 국가에서 RYTELO를 상용화할 계획이며, 이는 환급 승인을 기다리고 있습니다. 이 약물은 고아약 지정 상태를 유지하여 10년의 시장 독점권을 제공하며, 특허 독점권은 2038년까지 예상됩니다.
Geron (GERN) a reçu l'approbation de la Commission européenne (CE) pour RYTELO® (imetelstat), le premier inhibiteur de la télomérase de sa catégorie pour le traitement des adultes souffrant d'anémie dépendante des transfusions due à des MDS à faible risque (LR-MDS). L'approbation s'applique aux patients sans anomalie del-5q qui sont soit inéligibles, soit ont eu une réponse insatisfaisante à la thérapie à base d'érythropoïétine.
L'approbation est fondée sur les résultats de l'
L'autorisation de mise sur le marché de RYTELO couvre tous les 27 États membres de l'UE, l'Islande, la Norvège et le Liechtenstein. Geron prévoit de commercialiser RYTELO dans certains pays de l'UE à partir de 2026, sous réserve des approbations de remboursement. Le médicament conserve la désignation de médicament orphelin, offrant une exclusivité de marché de 10 ans, avec une exclusivité de brevet prévue jusqu'en 2038.
Geron (GERN) hat die Genehmigung der Europäischen Kommission (EK) für RYTELO® (imetelstat) erhalten, den ersten seiner Klasse von Telomerasehemmern zur Behandlung von Erwachsenen mit transfusionsabhängiger Anämie aufgrund von niedrig-risiko MDS (LR-MDS). Die Genehmigung gilt für Patienten ohne del-5q-Anomalie, die entweder nicht für die Therapie in Frage kommen oder eine unzureichende Reaktion auf erythropoietinhaltige Therapien hatten.
Die Genehmigung basiert auf den Ergebnissen der IMerge Phase 3-Studie, die eine signifikante Reduzierung der Abhängigkeit von roten Bluttransfusionen in den ersten 24 Wochen im Vergleich zu Placebo zeigte. Die häufigsten Nebenwirkungen umfassten Neutropenie (69%) und Thrombozytopenie (63%), die in der Regel innerhalb von vier Wochen abklingen.
Die Genehmigung für den Vertrieb von RYTELO umfasst alle 27 EU-Mitgliedstaaten, Island, Norwegen und Liechtenstein. Geron plant, RYTELO ab 2026 in ausgewählten EU-Ländern zu vermarkten, vorbehaltlich der Genehmigungen zur Kostenerstattung. Das Medikament behält den Status eines Orphan Drug und bietet eine 10-jährige Marktexklusivität, wobei die Patentschutzfrist bis 2038 erwartet wird.
- First-in-class telomerase inhibitor approved in both US and Europe
- Significant market exclusivity through orphan drug designation (10 years) and patent protection (until 2038)
- Phase 3 trial demonstrated significant reduction in transfusion dependency
- Expansion into European market increases commercial potential
- Commercialization in EU not starting until 2026
- High rates of serious adverse effects: neutropenia (69%) and thrombocytopenia (63%)
- Country-by-country reimbursement approval still pending
Insights
The European Commission approval of RYTELO (imetelstat) represents a significant milestone for Geron This first-in-class telomerase inhibitor now has regulatory approval in both the US and Europe, creating a substantial global commercial opportunity for the company's lead asset.
This approval specifically targets the treatment of adults with transfusion-dependent anemia due to lower-risk MDS without 5q deletion who failed or are ineligible for ESA therapy - a patient population with treatment options. The supporting IMerge Phase 3 trial demonstrated meaningful reduction in transfusion dependency, addressing a critical quality-of-life concern for these patients.
From a commercial perspective, this approval provides Geron access to 27 EU member states plus Iceland, Norway and Liechtenstein. While revenue generation will be delayed until 2026 pending country-by-country reimbursement negotiations, the company has secured strong intellectual property protection with anticipated patent exclusivity until 2038, plus 10 years of orphan drug market exclusivity.
The safety profile shows manageable and short-lived cytopenias, which are expected to be effectively managed by hematologists. This favorable benefit-risk profile should support adoption in clinical practice upon market entry.
This approval transforms Geron from a single-market to a global commercial-stage company with a novel therapeutic approach, representing a substantial expansion of their addressable market for a product with strong differentiation.
- Approval across ESA ineligible and ESA relapsed/refractory non-del 5q patients with transfusion-dependent anemia due to LR-MDS, regardless of ring sideroblast (RS) status
-
RYTELO is the first and only telomerase inhibitor approved in the
U.S. andEurope
Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today announced that the European Commission (EC) has granted marketing authorization for RYTELO® (imetelstat) as a monotherapy for the treatment of adult patients with transfusion-dependent (TD) anemia due to very low, low or intermediate risk myelodysplastic syndromes (lower-risk MDS or LR-MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy (ESAs). Lower-risk MDS is a progressive blood cancer with high unmet need, where many patients with anemia become dependent on red blood cell transfusions, which can be associated with clinical consequences and decreased quality of life.
“As the first and only treatment of its kind, RYTELO represents an important new option – significantly reducing the need for red blood cell transfusions for people living with LR-MDS who are battling debilitating symptoms like anemia and fatigue,” said Joseph Eid, M.D., Geron’s Executive Vice President, Research and Development. “This approval from the European Commission, just nine months following approval in the
“I am thrilled that the European Commission has approved RYTELO in LR-MDS. The long-term and durable responses observed in the Phase 3 IMerge study reinforce the practice-changing potential of telomerase inhibition as a clinically meaningful and differentiated option for the treatment of lower-risk MDS,” said Uwe Platzbecker, M.D., Chief Medical Officer at the University Hospital Carl Gustav Carus Dresden in
The marketing authorization of RYTELO approved by the EC is supported by data from the IMerge Phase 3 clinical trial, which demonstrated the significant clinical benefit of RYTELO in patients with transfusion-dependent anemia due to LR-MDS, reducing the need for red blood cell transfusions in the first 24 weeks of treatment compared to placebo, as observed in the double-blind controlled study. The safety profile of RYTELO was well-characterized with generally manageable and short-lived thrombocytopenia and neutropenia, which are familiar side effects for hematologists who are experienced with managing cytopenias. The most commonly reported adverse reactions ≥ Grade 3 were neutropenia (
RYTELO is the first and only telomerase inhibitor approved by the EC, and the marketing authorization applies to all 27 European Union member states, and
In connection with the approval, the EMA’s Committee of Orphan Medicinal Products (COMP) reviewed and issued a positive opinion to maintain RYTELO’s orphan drug designation in the EU for MDS, which is expected to provide market exclusivity for ten years after approval, subject to maintaining orphan designation. Patent exclusivity in the EU for LR-MDS is anticipated into 2038, subject to approval of patent term extension by the European Patent Office.
About Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue1. These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival2,3. There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis. Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence.
About RYTELO® (imetelstat)
RYTELO is an oligonucleotide telomerase inhibitor approved in the
In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.
RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the
WARNINGS AND PRECAUTIONS
Thrombocytopenia
RYTELO can cause thrombocytopenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased platelets occurred in
Monitor patients with thrombocytopenia for bleeding. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer platelet transfusions as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Neutropenia
RYTELO can cause neutropenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased neutrophils occurred in
Monitor patients with Grade 3 or 4 neutropenia for infections, including sepsis. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Infusion-Related Reactions
RYTELO can cause infusion-related reactions. In the clinical trial, infusion-related reactions occurred in
Premedicate patients at least 30 minutes prior to infusion with diphenhydramine and hydrocortisone as recommended and monitor patients for one hour following the infusion as recommended. Manage symptoms of infusion-related reactions with supportive care and infusion interruptions, decrease infusion rate, or permanently discontinue as recommended.
Embryo-Fetal Toxicity
RYTELO can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with RYTELO and for 1 week after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in
Most common adverse reactions (≥
Please see RYTELO (imetelstat) full Prescribing Information, available at https://pi.geron.com/products/US/pi/rytelo_pi.pdf.
The Summary of Product Characteristics (SmPC) for RYTELO in the EU is available at https://pi.geron.com/products/rytelo/eu/rytelo_smpc_eu.pdf
About Geron
Geron is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. Our first-in-class telomerase inhibitor RYTELO™ (imetelstat) is approved in
Use of Forward-Looking Statements
Except for the historical information contained herein, this press release contains forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such statements, include, without limitation, those regarding: (i) that lower-risk MDS is a progressive blood cancer with high unmet need, where many patients with anemia become dependent on red blood cell transfusions, which can be associated with clinical consequences and decreased quality of life; (ii) that RYTELO represents an important new option – significantly reducing the need for red blood cell transfusions for LR-MDS patients battling debilitating symptoms like anemia and fatigue, (iii) that approval of RYTELO in the European Union underscores the positive benefit demonstrated in Geron’s clinical trials and that Geron looks forward to making this innovative therapy accessible to eligible patients in
1 |
|
Lewis R, Bewersdorf JP, Zeidan AM. Clinical Management of Anemia in Patients with Myelodysplastic Syndromes: An Update on Emerging Therapeutic Options. Cancer Manag Res. 2021 Jan 25;13:645-657. doi: 10.2147/CMAR.S240600. PMID: 33531837; PMCID: PMC7846829. |
2 |
|
Cogle CR, Reddy SR, Chang E, et al. Early treatment initiation in lower-risk myelodysplastic syndromes produces an earlier and higher rate of transfusion independence. Leuk Res. 2017;60:123-128. |
3 |
|
Balducci, L. (2006), Transfusion independence in patients with myelodysplastic syndromes. Cancer, 106: 2087-2094. https://doi.org/10.1002/cncr.21860 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20250311485595/en/
Aron Feingold
Vice President, Investor Relations and Corporate Communications
Kristen Kelleher
Associate Director, Investor Relations and Corporate Communications
investor@geron.com
media@geron.com
Source: Geron Corporation
FAQ
What are the key clinical trial results for RYTELO (GERN) in treating lower-risk MDS?
When will GERN begin commercializing RYTELO in European markets?
What market exclusivity benefits does RYTELO (GERN) have in Europe?
Which patient population is eligible for RYTELO treatment in Europe?
