Rytelo (imetelstat) is a medicine used to treat myelodysplastic syndromes (MDS). MDS is a rare group of disorders that affect your blood cells. Most people who get MDS are 65 years of age or older, but younger people can get it too. With MDS, your bone marrow cells become abnormal and make blood cells that do not grow or work like normal cells. The syndromes are a type of cancer. Some cases are mild, and others may be serious. Symptoms include anemia, feeling very tired or weak, feeling short of breath when you are exercising or being active, bleeding, bruising, and paleness. Treatment depends on the type of MDS you have and how severe it is.
Treatment may include regular checkups and lab visits to check your blood cell counts, blood or platelet transfusions, or medicines called erythropoiesis-stimulating agents (ESAs) that increase the number of red blood cells in your body. Lifestyle changes are also helpful, such as not smoking, regular exercise, eating healthy foods, sleep, and stress management.
Rytelo is a medicine used for certain people with MDS that have low- to intermediate-1 risk MDS. It is approved to treat adults that have anemia, require red blood cell transfusions, and cannot take ESAs because these medicines are no longer working or cannot be taken due to other reasons.
How Does Rytelo Work for Myelodysplastic Syndromes?
The main ingredient in Rytelo is imetelstat, an anticancer medicine that blocks the activity of an enzyme called telomerase. Telomerase is more active in abnormal MDS cells. Rytelo binds to telomerase, which helps to stop more abnormal blood cells from forming and may cause the abnormal cells to die. This helps the bone marrow recover and make normal blood cells, which improves symptoms of anemia and reduces the need for blood transfusions.
How Is Rytelo Given?
Rytelo is given as an intravenous (IV) infusion through a needle placed in your vein by a health care provider. The infusion is given once every four weeks. Each infusion lasts about two hours. Rytelo can cause IV-related reactions and allergic reactions, so your health care provider will likely monitor you for these side effects during and for at least one hour after your infusion. Your health care provider may slow or stop your infusion if you have a reaction. You will likely be given medicine such as diphenhydramine (or another antihistamine) and hydrocortisone (or another steroid) by mouth or IV about 30 minutes before your Rytelo infusion to prevent or lessen reactions. Infusion-related reactions may include headache, feeling unusually tired, muscle or joint pain, back pain, stomach pain, diarrhea, itching, redness, or hives.
Your health care provider may change your dose of Rytelo, change your dosing schedule, or stop treatment depending on how you respond. You will need certain blood tests during treatment to check for side effects and to see how well Rytelo is working.
How Was Rytelo Studied for Myelodysplastic Syndromes?
The effectiveness and safety of Rytelo in adults with lower-risk MDS who require blood transfusions was studied in a clinical trial called IMerge.
Here are some details about the people in the study.
- All the people in the study had low or intermediate-1 risk MDS, were dependent on blood transfusions requiring four or more units of red blood cells over an eight-week period, and could not take ESAs because these agents had either not worked or stopped working, or they could not take ESA treatment because their blood erythropoietin concentrations were too high.
- The study included a total of 178 people, and the average age was 72 years.
- Most of the participants were male (62%), and the rest were female.
- Most were White (80%), 6% were Asian, 1.7% were Black or African American, and 12% identified their race as Other or did not report their race.
- Most people had low-risk MDS (67%) based on the International Prognostic Scoring System (IPSS), and 33% of the people had intermediate-1 risk disease.
- Most people received an average of six units of red blood cell transfusions over eight weeks, and 47% of people received more than six units of red blood cell transfusions over eight weeks.
- Almost all people (90%) had previously received an ESA, and 6% had previously received a medicine for MDS called luspatercept.
People in the study were treated with an IV infusion of Rytelo or a placebo (containing no medicine) every four weeks until their condition got worse, they had unacceptable side effects, or they wanted to leave the study. There were 118 people in the Rytelo group and 60 people in the placebo group. The study lasted about 18 months.
The main goal of the study was to look at the percentage of people who did not need blood transfusions for at least eight weeks or longer.
What Benefits of Rytelo Were Seen?
More people in the Rytelo group (40%) did not need a blood transfusion for eight consecutive weeks compared to the placebo group (15%). The typical time that these people did not need blood transfusions was 51.6 weeks in the Rytelo group compared to 13.3 weeks for people in the placebo group.
Some people did not need a blood transfusion for 24 consecutive weeks or more. There were 28% of people in the Rytelo group compared to 3% in the placebo group who did not require a blood transfusion for 24 or more weeks.
People who took Rytelo had greater improvement in their anemia and in symptoms of feeling tired compared to placebo. The average increase in hemoglobin, a protein in your red blood cells that carries oxygen, was 3.55 g/dL in the Rytelo group compared to 0.8 g/dL in the placebo group. More people in the Rytelo group (50%) had improvement in feeling tired compared to people in the placebo group (40%).
Your results may differ from what was seen in clinical studies.
How Long Does Rytelo Take to Work?
Rytelo may take several weeks to work to lower your need to have blood transfusions due to your anemia. In the study, the benefits of Rytelo were greater than placebo at eight weeks. Over time, people in the Rytelo group who responded continued to have greater increases in their hemoglobin levels and less blood transfusions than placebo. The time it took to have lasting improvement in feeling tired was 28 weeks for Rytelo compared to 65 weeks for placebo. Your health care provider will monitor you while you are receiving Rytelo. Your health care provider may adjust your dose or stop treatment with Rytelo depending on how well the medicine works.
Getting Your Medicine: What to Expect
You will receive Rytelo at an infusion center or in a hospital or clinic. The drugmaker has a program called the REACH4RYTELO Patient Support Program to help support the process for you and your health care provider. You can find out more at www.rytelo.com/patient-support or by calling 844-479-8356.
Insurance approval. Your insurance may require approval for using this medicine, also called a prior authorization. The insurance company reviews the prescription from your health care provider to make sure it is covered.
Cost support. There is a cost support program from the drugmaker that may allow you to pay $0 for your prescription. For questions or more information, contact the drugmaker at 844-479-8356.
