- 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. and Europe
FOSTER CITY, CA, USA I March 11, 2025 I 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 U.S., underscores the positive benefit for these patients demonstrated in our clinical trials and we look forward to making this innovative therapy accessible to eligible patients in Europe.”
“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 Germany, who was an IMerge investigator and a co-lead author of the Phase 3 results published in The Lancet. “Physicians and patients in Europe are now one step closer to accessing a novel treatment that, in addition to having a generally manageable safety profile, has the potential to provide extended and continuous red blood cell transfusion independence.”
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 (69%), thrombocytopenia (63%), which lasted a median duration of less than two weeks, and in more than 80% of patients were resolved to Grade < 2 in under four weeks.
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 Iceland, Norway and Liechtenstein. Geron is preparing to commercialize RYTELO in select EU countries beginning in 2026, pending country-by-country reimbursement. Additionally, Geron is exploring opportunities to make RYTELO available to eligible patients through Expanded Access Programs (EAP), including Named Patient Programs (NPP), which are designed to support access for individual patients on a case-by-case basis.
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 U.S. 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.
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 U.S. Food and Drug Administration and the European Commission.
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 the United States and the European Union for the treatment of certain adult patients with lower-risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia. We are also conducting a pivotal Phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis (R/R MF), as well as studies in other hematologic malignancies. Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit www.geron.com or follow us on LinkedIn.
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
SOURCE: Geron
Post Views: 136
- 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. and Europe
FOSTER CITY, CA, USA I March 11, 2025 I 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 U.S., underscores the positive benefit for these patients demonstrated in our clinical trials and we look forward to making this innovative therapy accessible to eligible patients in Europe.”
“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 Germany, who was an IMerge investigator and a co-lead author of the Phase 3 results published in The Lancet. “Physicians and patients in Europe are now one step closer to accessing a novel treatment that, in addition to having a generally manageable safety profile, has the potential to provide extended and continuous red blood cell transfusion independence.”
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 (69%), thrombocytopenia (63%), which lasted a median duration of less than two weeks, and in more than 80% of patients were resolved to Grade < 2 in under four weeks.
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 Iceland, Norway and Liechtenstein. Geron is preparing to commercialize RYTELO in select EU countries beginning in 2026, pending country-by-country reimbursement. Additionally, Geron is exploring opportunities to make RYTELO available to eligible patients through Expanded Access Programs (EAP), including Named Patient Programs (NPP), which are designed to support access for individual patients on a case-by-case basis.
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 U.S. 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.
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 U.S. Food and Drug Administration and the European Commission.
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 the United States and the European Union for the treatment of certain adult patients with lower-risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia. We are also conducting a pivotal Phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis (R/R MF), as well as studies in other hematologic malignancies. Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit www.geron.com or follow us on LinkedIn.
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
SOURCE: Geron
Post Views: 136
