ModifiedCentenePolicy N/A

Imetelstat (Rytelo) for transfusion-dependent myelodysplastic syndromes

Defines medical necessity criteria, prior authorization and coverage rules for imetelstat (Rytelo) for adult patients with transfusion-dependent myelodysplastic syndromes (MDS) across Centene lines of business.

Policy Summary

PayerCentene

PolicyImetelstat (Rytelo) for transfusion-dependent myelodysplastic syndromes

Policy CodePolicy N/A

Change TypeCoding and step therapy updates

Effective DateSep 1, 2024

Next Review DateN/A

Key ActionSubmit prior authorization showing diagnosis, transfusion history (≥4 RBC units/8 weeks), prior ESA failure/ineligibility, and prescriber specialty.

SourceLink

POLICY UPDATE CHANGES

HCPCS code J0870 (injection, imetelstat) was added and codes C9399 and J99991 were removed.

Step therapy bypass for Illinois HIM per IL HB 5395 (effective 1/1/2026) was added.

7.1 mg/kgMaximum FDA dosing for MDS indication

≥4 RBC/8wTransfusion dependence threshold

Adults ≥18Age requirement

J0870Current billing code

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

State overrideMedicaid precedence

Coverage and Medical Necessity Criteria

inv-01: Initial Therapy

Covered when ALL of the following are met:

Initial approval for imetelstat: 1) Diagnosis of myelodysplastic syndromes (MDS) with transfusion-dependent anemia; 2) Age >= 18 years; 3) Documentation of at least 4 RBC units transfused over 8 weeks; 4) Prescribed by or in consultation with a hematologist or oncologist; 5) Member does not have del(5q) cytogenetic abnormality; 6) Inadequate response to, loss of response to, or ineligible for erythropoiesis‑stimulating agent (ESA) therapy (e.g., epoetin alfa, darbepoetin); 7) Dose does not exceed 7.1 mg/kg every 4 weeks (unless a higher dose is supported by practice guidelines or peer‑reviewed literature and prescriber provides supporting evidence); 8) Imetelstat (Rytelo) is not prescribed concurrently with luspatercept (Reblozyl).<= 7.1 mg/kg every 4 weeks

Prescribed regimen must be FDA‑approved or recommended by NCCN.

inv-02: Continuation Therapy

Continued therapy covered when ONE of the following is met plus specific MDS conditions:

Continued therapy general: a) Member is currently receiving medication via the Centene benefit or has previously met initial approval criteria; b) Member is responding positively to therapy as evidenced by a decrease in RBC transfusion requirement; c) Member is enrolled in a state/product with continuity of care regulations (if applicable).Dose must remain <= 7.1 mg/kg every 4 weeks

If a dose increase is requested, the increase must be supported by practice guidelines or peer‑reviewed literature with prescriber documentation; imetelstat must not be prescribed concurrently with luspatercept (Reblozyl).

Non‑FDA approved indications that are not specifically addressed in this policy are excluded from coverage unless the provider submits sufficient documentation of efficacy and safety in accordance with applicable off‑label use policies (for example, CP.CPA.09 for commercial, HIM.PA.154 for Health Insurance Marketplace, or CP PMN.53 for Medicaid) or unless the member's evidence of coverage explicitly allows the use.

When state Medicaid coverage provisions conflict with the coverage provisions in this clinical policy, state Medicaid coverage provisions take precedence. Refer to the applicable state Medicaid manual for specific coverage rules that may override this policy.

Requests for uses not listed in this policy that lack appropriate off‑label evidence or documentation will be considered not medically necessary and are not authorized for coverage under this policy.

Regimen	Indication	Dose / Schedule	Coverage Status
Imetelstat (Rytelo) 7.1 mg/kg intravenous infusion over 2 hours every 4 weeks
Myelodysplastic syndromes (MDS) with transfusion-dependent anemia
7.1 mg/kg IV infusion administered over 2 hours every 4 weeks (maximum dose per cycle: 7.1 mg/kg)
Covered when medical necessity criteria are met (see policy criteria)

Billing and Coding

HCPCS codes referencedHCPCS

J0870	Injection, imetelstat, 1 mg
C9399	Previously referenced (removed)
J99991	Previously referenced (removed)

inv-07: Transfusion dependence — threshold requirement

Transfusion dependence threshold>= 4 RBC units over 8 weeks

Supporting sourceFDA indication and initial approval criteria require 4 or more RBC units over 8 weeks

Applies toAdult patients with transfusion‑dependent lower‑risk MDS

inv-08: Dose limit — Maximum FDA dosing for MDS indication

Maximum FDA dosing for MDS indication7.1 mg/kg every 4 weeks

Dosing regimen7.1 mg/kg IV infusion over 2 hours every 4 weeks

Dose constraint in approval criteria

Prior Authorization and Provider Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Rytelo (HCPCS J0870). Provider must demonstrate the member meets all initial approval criteria including diagnosis of transfusion‑dependent myelodysplastic syndromes (MDS), transfusion history, hematology/oncology involvement, age, cytogenetic status, and prior ESA/step‑therapy requirements or documented contraindication/clinical intolerance.

HCPCS: J0870 (Rytelo / imetelstat)
Initial approval requires documentation: diagnosis of MDS with transfusion‑dependent anemia; prescribed by or in consultation with a hematologist/oncologist; age ≥ 18 years; documentation of ≥4 RBC units transfused over 8 weeks; absence of del(5q) cytogenetic abnormality; inadequate response to or ineligible for ESA therapy unless contraindicated
Step therapy: prior use of specified ESAs (e.g., Retacrit, Epogen) is required unless contraindicated or unavailable; IL HIM statutory bypass applies per IL HB 5395 (effective 01/01/2026) where applicable

Documentation Required

Required Documentation

Positioning and Line of Therapy

inv-21: salvage

Salvage‑line use:

Salvage indication-specific: For adult patients with transfusion‑dependent lower‑risk MDS who required >= 4 RBC units over 8 weeks and who have failed, lost response to, or are ineligible for erythropoiesis‑stimulating agents (ESAs).

Indication aligns with FDA‑approved population for Rytelo; dosing per approved regimen (7.1 mg/kg IV every 4 weeks).

Clinical Background

Imetelstat (Rytelo) is an oligonucleotide telomerase inhibitor indicated for adults with transfusion‑dependent lower‑risk myelodysplastic syndromes (MDS). The approved indication specifies patients who required ≥ 4 RBC units over 8 weeks and who have inadequate response to, lost response to, or are ineligible for erythropoiesis‑stimulating agents (ESAs). The agent is administered intravenously every 4 weeks with a maximum dosing of 7.1 mg/kg every 4 weeks for the MDS indication.

Key Definitions and Abbreviations

inv-18: ESA definition — erythropoiesis-stimulating agent

DefinitionESA: erythropoiesis‑stimulating agent

ExamplesEpoetin alfa (Epogen/Procrit), darbepoetin (Aranesp), Retacrit

Role in policyUsed as prior therapy — members must have failed, lost response to, or be ineligible for ESA therapy to qualify for imetelstat

inv-19: RBC definition — red blood cell

RBC abbreviationRBC: red blood cell

Usage in criteriaTransfusion counts (RBC units) are used to determine transfusion‑dependence threshold

Contextual note

Policy Changes and Revision Log

2024-07-16policy_created

Clinical policy created.

2024-08-24pandt_approval

Policy P&T approval recorded.

2024-11-07hcpcs_updateLatest

HCPCS code J0870 (injection, imetelstat) added and codes C9399 and J99991 removed from coding references.

Policy Summary

PayerCentene

PolicyImetelstat (Rytelo) for transfusion-dependent myelodysplastic syndromes

Policy CodePolicy N/A

Change TypeCoding and step therapy updates

Effective DateSep 1, 2024

Next Review DateN/A

Key ActionSubmit prior authorization showing diagnosis, transfusion history (≥4 RBC units/8 weeks), prior ESA failure/ineligibility, and prescriber specialty.

SourceLink

On This Page

Dose does not exceed 7.1 mg/kg every 4 weeks unless supported by guidelines or peer‑reviewed literature

Provider must submit supporting clinical documentation with the prior authorization request. Acceptable documentation includes office chart notes, relevant laboratory results, transfusion records, consultation notes from a hematologist/oncologist, and any evidence supporting off‑label use when requested.

Include transfusion history (dates and number of RBC units transfused)
Include prior ESA treatment records or documentation of contraindication/intolerance
Include cytogenetic testing results (to confirm absence of del(5q))

Note

Coverage Documentation Linkage

Coverage determinations are guided by this medical necessity policy but remain subject to the member's evidence of coverage and applicable plan terms, conditions, exclusions, limitations, and state/federal requirements. This policy does not guarantee payment; verify member benefits for specific coverage and prior authorization submission requirements.

Decisions subject to the member's evidence of coverage, certificate of coverage, or contract terms
Provider should verify benefits and any plan‑specific prior authorization processes before administering therapy

Denial Risk

Non‑FDA Indications

Requests for indications not addressed in this policy (non‑FDA or off‑label uses) will be denied unless the prescriber submits sufficient peer‑reviewed evidence or documentation of efficacy and safety per the plan's off‑label use policies.

See applicable off‑label use policies (e.g., CP.CPA.09, HIM.PA.154, CP.PMN.53) for required evidence standards

Denial Risk

Unauthorized Use Risk

Unauthorized copying, distribution, or use of this clinical policy is prohibited. Providers, members, and their representatives are bound by the terms and conditions of the Health Plan when submitting requests or claims.

This clinical policy is the property of the Health Plan; submission of claims or requests indicates agreement to the policy terms

Referenced in FDA indication and appendices as the unit for transfusion requirement

inv-20: Clinical policy definition — guidance on medical necessity; not a contract or substitute for clinical judgment

PurposeProvides medical necessity guidance to assist in making coverage decisions and administering benefits

Not a contractThis clinical policy does not constitute a contract, guarantee of payment, or substitute for clinical judgment

Provider responsibilityProviders are expected to exercise professional medical judgment and are solely responsible for medical advice and treatment