Imetelstat
Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.
| ID | DRUG-IMETELSTAT |
|---|---|
| Type | Drug |
| Aliases | RyteloІметельстат |
| Status | reviewed 2026-04-25 | pending_clinical_signoff |
| Diseases | DIS-MDS-LR |
| Sources | SRC-ESMO-MDS-2021 SRC-NCCN-AML-2025 |
Drug Facts
| Class | First-in-class telomerase inhibitor (oligonucleotide) |
|---|---|
| Mechanism | 13-mer N3'→P5' thio-phosphoramidate oligonucleotide complementary to the RNA template region (hTR/TERC) of human telomerase. Binds and inhibits telomerase enzymatic activity in malignant cells with short telomeres + telomerase upregulation. Approved June 2024 for transfusion-dependent low-/intermediate-1-risk MDS post-ESA failure on basis of IMerge phase-3 trial. |
| Typical dosing | 7.1 mg/kg IV every 4 weeks (2-hour infusion). Dose modifications for cytopenia (the dominant toxicity). Treatment duration in IMerge: median ~33 weeks; continue until loss of TI / unacceptable toxicity / disease progression. |
| Ukraine registered | False |
| NSZU reimbursed | False |
| Ukraine last verified | 2026-04-27 |
Warnings
- Severe cytopenia (boxed): thrombocytopenia, neutropenia — bleeding + infection risk; mandatory dose modification + transfusion + G-CSF support
Notes
Pivotal: IMerge (Platzbecker et al., Lancet 2024) phase-3 — imetelstat 7.1 mg/kg IV q4wk vs placebo in transfusion-dependent LR-MDS post-ESA failure. Primary endpoint 8-week RBC TI: 39.8% vs 15.0% (p<0.001). Sustained 24-week TI: 28% vs 3.3%. FDA approval June 2024 (Rytelo). Cytopenia dominant toxicity — active management mandatory (transfusion support, dose modification, G-CSF for severe neutropenia). EMA approval pending as of 2026-04. Ukraine: NOT registered — access only via named-patient import (after EMA approval) or US compassionate-use program; realistically unavailable in MVP timeframe.
Used By
Regimens
REG-IMETELSTAT-MDS-LR- Imetelstat (Rytelo) for transfusion-dependent LR-MDS post-ESA failure