RYTELO (imetelstat sodium)
Rytelo treats adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who suffer from anemia requiring frequent red blood cell transfusions. It is used for patients who need at least four units of blood over an eight-week period and have not seen results from erythropoiesis-stimulating agents. This medication helps patients who are either ineligible for these standard therapies or have lost their initial response to them.
How RYTELO Works
This drug works by binding to the RNA component of human telomerase to inhibit its enzymatic activity and prevent telomere binding. By blocking this enzyme, which is often overactive in MDS, the treatment reduces telomere length and slows the growth of malignant stem and progenitor cells. This process ultimately helps trigger the death of these diseased cells.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2024-06-06
- Patent Cliff
- 2039
- Routes
- INTRAVENOUS
- Dosage Forms
- POWDER
RYTELO Approval History
What RYTELO Treats
2 indicationsRYTELO is approved for 2 conditions since its original approval in 2024. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Myelodysplastic Syndromes
- Anemia
RYTELO Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in RYTELO's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications RYTELO treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to RYTELO
3 of 14FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
7 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05583552 IMpress results posted | IMpress_001 | Ph 2 | active not recruiting | Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy |
| NCT05371964 | MYF1001 | Ph 1 | recruiting | A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Participants With Myelofibrosis |
| NCT02598661 results posted | CR107947 63935937MDS3001, EU CTIS number | Ph 2, Ph 3 | active not recruiting | Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) |
| NCT02011126 | ADVL1321 NCI-2013-01640, ADVL1321 | Ph 2 | withdrawn | Imetelstat Sodium in Treating Younger Patients With Relapsed or Refractory Solid Tumors |
| NCT01836549 results posted | PBTC-036 NCI-2013-00482, U01CA081457 | Ph 2 | terminated | Imetelstat Sodium in Treating Younger Patients With Recurrent or Refractory Brain Tumors |
| NCT01256762 | CP14B014 | Ph 2 | completed | Imetelstat in Combination With Paclitaxel (With or Without Bevacizumab) in Patients With Locally Recurrent or Metastatic Breast Cancer |
| NCT01273090 | ADVL1112 COG-ADVL1112 | Ph 1 | completed | Imetelstat Sodium in Treating Young Patients With Refractory or Recurrent Solid Tumors or Lymphoma |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
RYTELO FDA Label Details
Indications & Usage
FDA Label (PDF)RYTELO is indicated for the treatment of Myelodysplastic Syndromes; Anemia.
RYTELO Patents & Exclusivity
Patents (6 active)
Exclusivity
Pro Intelligence Preview
Deep insights for RYTELO
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2039
- • 24 active patents
Trial Analysis
- • 7 total trials
- • Stage: Stable
Competitive Landscape
- • 14 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment