Drug updated on 10/18/2024
| Dosage Form | Injection (intravenous; 7.1 mg/Kg) |
| Drug Class | Oligonucleotide telomerase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- 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).
Latest News
Summary
- This summary is based on the review of two randomized controlled trial(s). [1-2]
- In the Phase 3 trial, 40% (47/118) of patients in the imetelstat group achieved 8-week RBC (ESA) transfusion independence (RBC-TI (Transfusion Independence)), compared to 15% (9/60) in the placebo group, with a rate difference of 25% (95% CI (Confidence Interval): 9.9 to 36.9; p=0.0008).
- In the Phase 2 trial, 23% of the overall population achieved 24-week RBC-TI, with a higher rate of 29% observed in the non-del(5q) and hypomethylating agent and lenalidomide naive subset. The median TI duration was 65 weeks for the overall population and 86 weeks for the subset.
- The hematologic improvement-erythroid was also evaluated in the Phase 2 trial, but specific results were not provided.
- In the Phase 3 trial, 91% (107/118) of patients in the imetelstat group experienced Grade 3-4 treatment-emergent adverse events, compared to 47% (28/59) in the placebo group. Common Grade 3-4 adverse events included neutropenia (68% imetelstat vs. 3% placebo) and thrombocytopenia (62% imetelstat vs. 8% placebo), with no treatment-related deaths reported and most cytopenias being reversible within 4 weeks.
- The population includes patients aged ≥18 years with ESA-relapsed, ESA-refractory, or ESA-ineligible lower-risk myelodysplastic syndromes (LR-MDS (low or intermediate-1 risk disease as per International Prognostic Scoring System [IPSS] criteria)). Subgroup analysis from the Phase 2 trial showed higher RBC transfusion independence (RBC-TI) rates in the non-del(5q) and hypomethylating agent and lenalidomide naive populations compared to the overall population. No unique safety concerns were identified for these subgroups beyond common reversible cytopenias.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Rytelo (imetelstat) Prescribing Information. | 2024 | Geron Corporation, Foster City, CA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial | Data not availableSubjects F: null% M: null% | 2024 | The Lancet |
| Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion-Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study | 57Subjects F: 44% M: 56% | 2021 | Journal of Clinical Oncology |
Sex Distribution:
Year:
2024
Source:The Lancet
Sex Distribution:
F:44%
M:56%
57Subjects
Year:
2021
Source:Journal of Clinical Oncology