Last Review Date: 07/02/2024

Date of Origin: 07/02/2024

Dates Reviewed: 07/2024

1. Length of Authorization ¹

• Coverage will be provided for 6 months and may be renewed.

2. Dosing Limits

1. Quantity Limit (max daily dose) [NDC Unit]:

• Rytelo 47 mg powder in a single-dose vial: 3 vials every 4 weeks
• Rytelo 188 mg powder in a single-dose vial: 5 vials every 4 weeks

2. Max Units (per dose and over time) [HCPCS Unit]:

• 940 mg every 4 weeks

3. Initial Approval Criteria ¹

     Coverage is provided in the following conditions:

• Patient is at least 18 years of age; AND

        Myelodysplastic Syndrome (MDS)

• Patient has IPSS symptomatic low- to intermediate-1-risk disease; AND
• Patient is relapsed or refractory to ESA therapy or is ESA ineligible (i.e., EPO>500 mU/mL); AND
• Patient is red blood cell (RBC) transfusion dependent, defined as requiring at least 4 RBC units transfused over an 8-week period

(Note: Patients with a deletion (5q) cytogenetic mutation, or who have previously received treatment with lenalidomide or a hypomethylating agent (e.g., azacitidine, decitabine, etc.), will be reviewed on a case-by-case basis.)

† FDA Approved Indications; ‡ Compendia Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ^1,5-8,12
   • Patient continues to meet universal and other indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in section III; AND

• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: severe thrombocytopenia, neutropenia, and severe infusion-related reactions, etc.; AND

Myelodysplastic Syndromes (MDS)

• • For first renewal: Patient has achieved a reduction in RBC transfusion burden after at least 24 weeks, (6 doses), from baseline; OR
  • For subsequent renewals: Patient is experiencing disease response as evidenced by a decrease in the number of RBC transfusions from baseline

5. Dosage/Administration ^1,12

6. Billing Code/Availability Information

HCPCS Code:

• J9999 – Not otherwise classified, antineoplastic drugs

NDC(s):

• Rytelo 47 mg powder in a single-dose vial: 82959-0112-xx
• Rytelo 188 mg powder in a single-dose vial: 82959-0111-xx

7. References

1. Rytelo [package insert]. Foster City, CA; Geron, Inc: June 2024. Accessed June 2024.
2. Referenced with permission from the NCCN Drugs & Biologics Compendium (NCCN Compendium^®) imetelstat. National Comprehensive Cancer Network, 2024. The NCCN Compendium^® is a derivative work of the NCCN Guidelines^®. NATIONAL COMPREHENSIVE CANCER NETWORK^®, NCCN^®, and NCCN GUIDELINES^® are trademarks owned by the National Comprehensive Cancer Network, Inc. To view the most recent and complete version of the Compendium, go online to NCCN.org. Accessed June 2024.
3. Referenced with permission from the NCCN Drugs & Biologics Compendium (NCCN Compendium^®) Myelodysplastic Syndromes. Version 1.2024. National Comprehensive Cancer Network, 2024. The NCCN Compendium^® is a derivative work of the NCCN Guidelines^®. NATIONAL COMPREHENSIVE CANCER NETWORK^®, NCCN^®, and NCCN GUIDELINES® are trademarks owned by the National Comprehensive Cancer Network, Inc. To view the most recent and complete version of the Compendium, go online to NCCN.org. Accessed June 2024.
4. Zeidan AM, Platzbecker U, Santini V, et al. IMerge: Results from a phase 3, randomized, double-blind, placebo-controlled study of imetelstat in patients (pts) with heavily transfusion dependent (TD) non-del(5q) lower-risk myelodysplastic syndromes (LR-MDS) relapsed/refractory (R/R) to erythropoiesis stimulating agents (ESA). Meeting Abstract: 2023 ASCO Annual Meeting I. Journal of Clinical Oncology Volume 41, Number 16_suppl June 2023.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCD/LCA): N/A