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INDICATION

RYTELO® (imetelstat) is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA). See more

Imetelstat (RYTELO®) NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®)
recommendations

The recommendations for Management of Lower-Risk MDS Disease, Treatment of Symptomatic Anemia, include imetelstat (RYTELO®) as an NCCN Category 1* and Category 2A† preferred treatment option for certain patients1,‡

Important disclosures

  • RYTELO (imetelstat) is an oligonucleotide telomerase inhibitor indicated for2:
    • Treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA)
  • NCCN Guidelines® for LR-MDS may describe certain uses of imetelstat that are not approved by the FDA. The safety and efficacy of any unapproved uses have not been established
  • The RYTELO (imetelstat) Prescribing Information includes warnings and precautions for the following adverse reactions: thrombocytopenia, neutropenia, infusion-related reactions, and embryo-fetal toxicity2
  • RYTELO (imetelstat) can cause serious adverse reactions including sepsis, fracture, cardiac failure, and hemorrhage, as well as fatal adverse reactions, including sepsis2

NCCN Guidelines recommend imetelstat (RYTELO®)1,‡

Table showing NCCN guidelines recommendations for RYTELO based on first and second line patient types.
Table showing NCCN guidelines recommendations for RYTELO based on first and second line patient types.

Note: All category recommendations are Category 2A unless otherwise indicated.†

1L, first line; 2L, second line; IPSS-R, Revised International Prognostic Scoring System; IST, immunosuppressive therapy; LR-MDS, lower-risk myelodysplastic syndromes; NCCN, National Comprehensive Cancer Network; RS, ring sideroblasts; sEPO, serum erythropoietin.

*Category 1: Based upon high-level evidence (≥1 randomized phase 3 trials or high-quality, robust meta-analyses), there is uniform NCCN consensus (≥85% support of the Panel) that the intervention is appropriate.1
†Category 2A: Based upon lower-level evidence, there is uniform NCCN consensus (≥85% support of the Panel) that the intervention is appropriate.1
‡For patients with IPSS-R very low-, low-, or intermediate-risk MDS and symptomatic anemia with non-del(5q) ± other cytogenetic abnormalities and with RS <15% (or RS <5% with an SF3B1 mutation) or with non-del(5q) ± other cytogenetic abnormalities with RS ≥15% (or RS ≥5% with an SF3B1 mutation).1
§Other interventions that may be somewhat less efficacious, more toxic, or based on less mature data, or significantly less affordable or similar outcomes.1

References: 1. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Myelodysplastic Syndromes V.2.2025. © National Comprehensive Cancer Network, Inc. 2025. All rights reserved. Accessed April 8, 2025. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 2. RYTELO. Prescribing information. Geron Corp.; 2024.

INDICATION

RYTELO® (imetelstat) is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

IMPORTANT SAFETY INFORMATION

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 65% of patients with MDS treated with RYTELO.

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 72% of patients with MDS treated with RYTELO.

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 8% of patients with MDS treated with RYTELO; Grade 3 or 4 infusion-related reactions occurred in 1.7%, including hypertensive crisis (0.8%). The most common infusion-related reaction was headache (4.2%). Infusion-related reactions usually occur during or shortly after the end of the infusion.

Premedicate patients at least 30 minutes prior to infusion with diphenhydramine and hydrocortisone as recommended and monitor patients for at least 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

Based on animal findings, 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 32% of patients who received RYTELO. Serious adverse reactions in >2% of patients included sepsis (4.2%), fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients who received RYTELO, including sepsis (0.8%).

Most common adverse reactions (≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets, decreased white blood cells, decreased neutrophils, increased AST, increased alkaline phosphatase, increased ALT, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.

Please see full Prescribing Information, including Medication Guide.

You are encouraged to report adverse events related to Geron products by calling 1-855-437-6664 (1-855-GERON-MI) (US only). If you prefer, you may contact the US Food and Drug Administration (FDA) directly. Visit www.fda.gov/MedWatch or call 1-800-FDA-1088.

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C-US-RYT-IM-0011 June 2025 © 2025 Geron Corp. All rights reserved. Geron, Geron logo, RYTELO, RYTELO logo, REACH4RYTELO, and the REACH4RYTELO logo are trademarks of Geron Corporation. All other trademarks are the property of their respective owners.