Rytelo Approved for Myelodysplastic Syndromes

On June 06, 2024, the U.S. Food and Drug Administration (FDA) approved Rytelo^™ (imetelstat – Geron Corporation). It’s an oligonucleotide telomerase inhibitor for treating adults with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell (RBC) units over eight weeks and have not responded to, or are ineligible for, erythropoiesis-stimulating agents (ESA), such Epogen^® (epoetin alfa). Rytelo is administered intravenously (IV) at a dose of 7.1mg/kg of body weight once every four weeks. The company plans to launch Rytelo by the end of June, with a wholesale acquisition cost (WAC) of $9,884 for the 188mg vial, and $2,471 for the 47mg vial. Full prescribing information is here.

At a Glance

• Brand Drug: Rytelo (imetelstat)
• Manufacturer: Geron Corporation 
• Date Approved: June 06, 2024
• Indication: for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs).
• Dosage Forms Available: single-dose vials containing 47mg or 188mg of powder for reconstitution and IV infusion
• Launch Date: By the end of June 2024
• Estimated Annual Cost: $340,000 (WAC) per year for a patient weighing 70 kg (154 pounds)
• Myelodysplastic syndromes (MDS) are a form of cancer that can develop when the blood-forming cells in the bone marrow become abnormal, resulting in a low count for one or more types of blood cells. It affects at least 10,000 Americans each year. While uncommon before age 50, its risk increases, with age with most patients diagnosed in their 70s.
• Rytelo is known as a telomerase inhibitor, which blocks an enzyme that allows for the uncontrolled division of the cancerous cells in the bone marrow that cause MDS, resulting in anemia.
• In the IMerge clinical trial, about 40% of patients treated with Rytelo did not require red blood cell transfusions for at least eight consecutive weeks, compared to 15% of those who received a placebo. In addition, 28% of Rytelo-treated patients did not require transfusions for at least 24 consecutive weeks, compared to only 3.3% of patients receiving the placebo. These effects lasted about one year and 1.5 years for the 8-week and 24-week responders, respectively.
• The most common adverse events (AEs) include laboratory abnormalities such as low platelets, white blood cells, neutrophils, and increased liver enzymes (AST, ALP, and ALT). As a result, complete blood cell counts should be evaluated before starting therapy with Rytelo, weekly for the first two cycles, and at least before each cycle after that.
• Rytelo was granted orphan drug designation.