Imetelstat Greenlighted for Some MDS Cases Free

The FDA approved imetelstat (Rytelo; Geron), an oligonucleotide telomerase inhibitor, for some adults with low- to intermediate-1–risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia. Efficacy was assessed in IMerge, a double-blind trial involving 178 patients with MDS who were assigned in a 2:1 ratio to receive either imetelstat or a placebo. After a median follow-up of 19.5 months in the imetelstat group and 17.5 months in the placebo group, 39.8% and 15% of patients, respectively, did not require a red blood cell transfusion during any 8-week period; 28% and 3.3%, respectively, did not need a transfusion during any 24-week period. The most common side effects were decreases in platelets, white blood cells, and neutrophils.