Approval Date: June 6, 2024
Trade Name (Generic Name): Rytelo™ (imetelstat)
Approved For: Treatment of adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia.
Eligibility: Patients requiring four or more red blood cell units over eight weeks who have not responded to, lost response to, or are ineligible for erythropoiesis-stimulating agents (ESAs).
Clinical Trial & Efficacy
IMerge Phase 3 Trial:
Primary Endpoint: Red blood cell transfusion independence (RBC-TI) for at least 8 weeks and 24 weeks.
Results:
8 Weeks: Rytelo 39.8% vs. Placebo 15.0%
24 Weeks: Rytelo 28.0% vs. Placebo 3.3%
Hemoglobin Increase: 3.6 g/dL for Rytelo vs. 0.8 g/dL for placebo.
Safety: Generally manageable thrombocytopenia and neutropenia.
Benefits
Transfusion Independence: Potential for over 24 weeks.
Improved Hemoglobin Levels: Clinically significant increases.
Quality of Life: Reduction in anemia-related symptoms and improved fatigue.
Safety Information
Common Adverse Reactions (Grade 3/4): Neutropenia (72%) and thrombocytopenia (65%).
Adverse Reaction Duration: Typically resolved within four weeks.
Other Adverse Reactions: Increased liver enzymes, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, infections (including COVID-19), and headache.
Serious Adverse Reactions: Sepsis (4.2%), fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%).
Administration
Method: Intravenous infusion.
Frequency: Every four weeks.
Special Precautions
Monitoring Needed: Complete blood cell counts, infection signs, and potential infusion-related reactions.
Embryo-Fetal Toxicity: Possible risks; women of reproductive potential should use effective contraception