Imetelstat received U.S. Food and Drug Administration (FDA) approval for the treatment of low- to intermediate-1 risk myelodysplastic syndromes (MDS) in adults with transfusion-dependent anemia who require four or more red blood cell (RBC) units over eight weeks and have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents.
Imetelstat is an oligonucleotide telomerase inhibitor that was evaluated in a randomized, double-blind, placebo-controlled, multicenter trial dubbed IMerge. Results found an eight-week or greater RBC transfusion independence rate of 39.8% compared to 15% in the placebo arm.
Adverse reactions observed included arthralgia/myalgia, decreased platelets, COVID-19 infection, headache, decreased neutrophils, increased alkaline phosphatase, prolonged partial thromboplastin time, fatigue, decreased white blood cells, increased aspartate aminotransferase, increased alkaline phosphatase, and increased alanine aminotransferase.
“For patients with lower-risk MDS and anemia who are transfusion dependent, we have very few options today and often cycle through available therapies,” said Rami Komrokji, MD, vice chair of the Malignant Hematology Department at Moffitt Cancer Center in Tampa, Florida, and investigator of IMerge. “What is exciting about [imetelstat] is the totality of the clinical benefit across patients with [lower-risk] MDS irrespective of ring sideroblast status or high transfusion burden, including sustained and durable transfusion independence and increases in hemoglobin levels, all within a well-characterized safety profile of generally manageable cytopenias. The treatment goal for patients with [lower-risk] MDS and anemia is transfusion independence, and before today, this wasn’t possible for many patients.”
Sources: FDA, June 6, 2024. Geron, June 6, 2024.
