The US Food and Drug Administration has approved imetelstat, an oligonucleotide telomerase inhibitor, for adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have transfusion-dependent anemia and require four or more units of red blood cells over 8 weeks, and who have not responded to or lost response to, or are ineligible for erythropoiesis-stimulating agents. Clinical trial The efficacy of imetelstat was evaluated in the IMerge trial (NCT02598661), a randomized, double-blind, placebo-controlled multicenter study involving 178 MDS patients.
Participants received either imetelstat (7.1 mg/kg) or placebo via intravenous infusion in 28-day cycles until disease progression or unacceptable toxicity, with randomization based on prior RBC transfusion burden and International Prognostic Scoring System risk group. The imetelstat group showed a significant improvement,…
