What is it prescribed for?
RYTELO, approved on June 6, 2024, is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).
What is the name of the drug and what does it do?
Rytelo (pronounced ri-TEL-o), (generic name: imetelstat), is a medication indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have transfusion-dependent anemia.
This condition requires patients to receive 4 or more red blood cell units over 8 weeks and who have not responded to, or have lost response to, erythropoiesis-stimulating agents (ESA), or are ineligible for such treatment.
How does it work?
Rytelo is an oligonucleotide telomerase inhibitor. Telomerase is an enzyme that helps maintain the length of telomeres, which are protective caps at the ends of chromosomes.
In many cancer cells, including those in MDS, telomerase is overactive, allowing cells to keep dividing uncontrollably.
Rytelo binds to the RNA component of human telomerase, inhibiting its activity and preventing the maintenance of telomeres.
This inhibition leads to reduced proliferation and increased death of malignant stem and progenitor cells, which are responsible for the disease.
What did the research discover?
Clinical trials, including the IMerge study, have shown that Rytelo effectively reduces the need for red blood cell transfusions in patients with MDS.
The trials demonstrated that a higher proportion of patients treated with Rytelo achieved transfusion independence for at least 8 weeks compared to those receiving a placebo.
Additionally, many patients maintained this independence for 24 weeks or more, indicating a significant improvement in managing the disease.
What are some of the side effects?
- Decreased platelet counts
- Decreased white blood cell counts
- Decreased neutrophil counts
- Increased AST (aspartate aminotransferase)
- Increased alkaline phosphatase
- Increased ALT (alanine aminotransferase)
- Fatigue
- Prolonged partial thromboplastin time (PTT)
- Arthralgia/myalgia (joint and muscle pain)
- COVID-19 infections
- Headache
What are the dosage recommendations and how is it prescribed?
Rytelo is administered as an intravenous infusion. The recommended dosage is 7.1 mg/kg given over 2 hours every 4 weeks.
Premedication with diphenhydramine and hydrocortisone is advised to reduce the risk of infusion-related reactions. Dosage adjustments or discontinuation may be necessary based on the patient’s response and the occurrence of adverse reactions.
Monitoring of blood cell counts is essential before each cycle and weekly for the first two cycles.
Source:
- Geron Corporation. Rytelo (imetelstat) for injection, for intravenous use. Prescribing Information. 2024. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/217779s000lbl.pdf
