Geron Announces Positive CHMP Opinion for RYTELO™ (imetelstat) for the Treatment of Adults with Transfusion-Dependent Anemia due to Lower-Risk MDS
Geron (Nasdaq: GERN) received a positive opinion from the European Medicines Agency's CHMP recommending the approval of RYTELO (imetelstat) for treating adult patients with transfusion-dependent anemia due to lower-risk myelodysplastic syndromes (LR-MDS). The treatment is specifically for patients without an isolated deletion 5q cytogenetic abnormality who had unsatisfactory response to or are ineligible for erythropoietin-based therapy.
The recommendation is based on the IMerge Phase 3 clinical trial results, which demonstrated reduced need for red blood cell transfusions in the first 24 weeks compared to placebo. If approved by the European Commission, RYTELO would become the first telomerase inhibitor available in Europe, administered as 47 mg and 188 mg lyophilized powder for intravenous infusion.
Geron (Nasdaq: GERN) ha ricevuto un parere positivo dal CHMP dell'Agenzia Europea dei Medicinali per raccomandare l'approvazione di RYTELO (imetelstat) per il trattamento di pazienti adulti con anemia da trasfusione dipendente a causa di sindromi mielodisplastiche a basso rischio (LR-MDS). Questo trattamento è specificamente per pazienti senza un'anormalità citogenetica isolata di delezione 5q che hanno avuto una risposta insoddisfacente o che non sono idonei alla terapia a base di eritropoietina.
La raccomandazione si basa sui risultati dello studio clinico di Fase 3 IMerge, che ha dimostrato una riduzione della necessità di trasfusioni di globuli rossi nelle prime 24 settimane rispetto al placebo. Se approvato dalla Commissione Europea, RYTELO diventerebbe il primo inibitore della telomerasi disponibile in Europa, somministrato come polvere liofilizzata da 47 mg e 188 mg per infusione endovenosa.
Geron (Nasdaq: GERN) recibió una opinión positiva del CHMP de la Agencia Europea de Medicamentos recomendando la aprobación de RYTELO (imetelstat) para el tratamiento de pacientes adultos con anemia dependiente de transfusiones debido a síndromes mielodisplásticos de bajo riesgo (LR-MDS). Este tratamiento es específicamente para pacientes sin una anormalidad citogenética aislada de deleción 5q que tuvieron una respuesta insatisfactoria o que no son elegibles para la terapia basada en eritropoyetina.
La recomendación se basa en los resultados del ensayo clínico de Fase 3 IMerge, que demostró una reducción en la necesidad de transfusiones de glóbulos rojos en las primeras 24 semanas en comparación con el placebo. Si es aprobado por la Comisión Europea, RYTELO se convertiría en el primer inhibidor de la telomerasa disponible en Europa, administrado como polvo liofilizado de 47 mg y 188 mg para infusión intravenosa.
제론 (Nasdaq: GERN)은 유럽 의약품청(CHMP)으로부터 저위험 골수형성이상 증후군(LR-MDS)으로 인한 수혈 의존성 빈혈 치료를 위한 RYTELO(imetelstat) 승인을 권장하는 긍정적인 의견을 받았습니다. 이 치료는 5q의 고립된 결실 세포 유전적 이상이 없는 환자와 erythropoietin 기반 요법에 대한 불만족스러운 반응을 보였거나 자격이 없는 환자에게 특히 해당됩니다.
이 권장은 24주 동안 위약 대비 적혈구 수혈 필요성을 줄인 IMerge 3상 임상 시험 결과를 바탕으로 합니다. 유럽연합 집행위원회에서 승인될 경우, RYTELO는 유럽에서 처음으로 사용할 수 있는 텔로머라 제 억제제가 되며, 47mg 및 188mg의 리오필라이즈 분말로 정맥 주사로 투여됩니다.
Geron (Nasdaq: GERN) a reçu un avis positif de la CHMP de l'Agence européenne des médicaments recommandant l'approbation de RYTELO (imetelstat) pour le traitement des patients adultes atteints d'anémie dépendante des transfusions due à des syndromes myélodysplasiques à faible risque (LR-MDS). Ce traitement est spécifiquement destiné aux patients sans anomalie cytogénétique isolée de délétion 5q qui ont eu une réponse insatisfaisante ou qui ne sont pas éligibles à la thérapie à base d'érythropoïétine.
La recommandation est basée sur les résultats de l'essai clinique de phase 3 IMerge, qui a démontré une réduction du besoin en transfusions de globules rouges au cours des 24 premières semaines par rapport au placebo. Si approuvé par la Commission européenne, RYTELO deviendrait le premier inhibiteur de la télomérase disponible en Europe, administré sous forme de poudre lyophilisée de 47 mg et 188 mg pour infusion intraveineuse.
Geron (Nasdaq: GERN) erhielt eine positive Stellungnahme von dem CHMP der Europäischen Arzneimittel-Agentur, die die Genehmigung von RYTELO (imetelstat) zur Behandlung von erwachsenen Patienten mit transfusionsabhängiger Anämie aufgrund von myelodysplastischen Syndromen mit geringem Risiko (LR-MDS) empfiehlt. Die Behandlung richtet sich speziell an Patienten ohne eine isolierte Deletion 5q zytogenetische Anomalie, die unzureichend auf eine Therapie mit Erythropoietin reagiert haben oder dafür nicht in Frage kommen.
Die Empfehlung basiert auf den Ergebnissen der IMerge Phase-3-Studie, die in den ersten 24 Wochen im Vergleich zu Placebo einen reduzierten Bedarf an Bluttransfusionen zeigte. Wenn die Europäische Kommission genehmigt, würde RYTELO der erste in Europa verfügbare Telomerasehemmer werden, der als 47 mg und 188 mg gefriergetrocknetes Pulver für die intravenöse Infusion verabreicht wird.
- Received positive CHMP recommendation for RYTELO approval in EU
- Demonstrated efficacy in reducing blood transfusion dependency in Phase 3 trial
- Would be first-in-class telomerase inhibitor in Europe if approved
- Treatment requires regular monitoring by healthcare professionals
- Multiple common side effects reported including thrombocytopenia and leukopenia
Insights
The CHMP's positive opinion for RYTELO represents a significant regulatory milestone for Geron. The recommendation for approval in treating transfusion-dependent anemia in lower-risk MDS patients is based on compelling Phase 3 IMerge trial data. As the first potential telomerase inhibitor in Europe, RYTELO would address a critical unmet need for patients who have treatment options after failing erythropoietin therapy. The drug's ability to reduce red blood cell transfusion dependency could significantly improve patient quality of life and reduce healthcare burden.
The safety profile appears manageable, with expected hematologic adverse events that can be monitored. If approved by the EC, this would mark Geron's entry into the European market, potentially capturing a substantial portion of the MDS treatment landscape. The specialized administration requirements under experienced healthcare professionals suggest a controlled distribution strategy that could support premium pricing.
This regulatory advancement significantly strengthens Geron's commercial prospects in Europe. With a
Market penetration in select EU countries could provide meaningful revenue streams, complementing U.S. opportunities. The requirement for specialized administration suggests a focused commercial strategy targeting hematology centers, potentially optimizing launch costs while maintaining pricing power. This milestone reduces regulatory risk and enhances Geron's market position in hematologic therapeutics.
“The positive CHMP opinion is an important step towards our goal to optimize value and patient access to RYTELO in the European Union, where we look forward to the opportunity to make this important new treatment option for LR-MDS patients available in select markets. If approved, RYTELO would be the first and only telomerase inhibitor available in Europe,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “We are deeply appreciative to the patients, caregivers, advocates and investigators across the EU who contributed to the clinical development of RYTELO.”
As part of its review of the marketing authorization application (MAA), the CHMP looked at the results from the IMerge Phase 3 clinical trial and assessed that the benefit of RYTELO in patients with transfusion-dependent anemia due to very low, low or intermediate risk MDS is a reduction in 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 most common side effects were thrombocytopenia, leukopenia, neutropenia, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), increased alkaline phosphatase (ALP), asthenia and headache.
If approved by the EC, RYTELO will be available as 47 mg and 188 mg lyophilized powder for reconstitution in a solution that is administered as an intravenous infusion. Treatment with RYTELO should be administered and monitored under the supervision of physicians and healthcare professionals who are experienced in hematologic diseases and their treatment.
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)
A marketing authorization application for RYTELO™ (imetelstat) is under review by the European Commission as a monotherapy treatment for adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes (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.
RYTELO is an FDA-approved oligonucleotide telomerase inhibitor for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.
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, including Medication Guide, available at https://pi.geron.com/products/US/pi/rytelo_pi.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) the EC’s review of the CHMP’s recommendation and expected timing of its decision on the MAA for RYTELO; (ii) Geron’s goal to optimize value and patient access to RYTELO in the European Union and its plans to make RYTELO available in select markets, subject to regulatory approval; and (iii) other statements that are not historical facts, constitute forward-looking statements. These forward-looking statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation, risks and uncertainties related to: (a) whether Geron is successful in commercializing RYTELO (imetelstat) for the treatment of certain patients with LR-MDS with transfusion dependent anemia; (b) whether Geron overcomes potential delays and other adverse impacts caused by enrollment, clinical, safety, efficacy, technical, scientific, intellectual property, manufacturing and regulatory challenges in order to have the financial resources for and meet expected timelines and planned milestones; (c) whether regulatory authorities permit the further development of imetelstat on a timely basis, or at all, without any clinical holds; (d) whether any future safety or efficacy results of imetelstat treatment cause the benefit-risk profile of imetelstat to become unacceptable; (e) whether imetelstat actually demonstrates disease-modifying activity in patients and the ability to target the malignant stem and progenitor cells of the underlying disease; (f) that Geron may seek to raise substantial additional capital in order to continue the development and commercialization of imetelstat; (g) whether Geron meets its post-marketing requirements and commitments in the
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
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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
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