FDA approves first-in-class treatment for low-risk MDS with transfusion-dependent anemia

The US Food and Drug Administration (FDA) approved imetelstat for adults with low- to intermediate-1-risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia who require 4 of more red blood cell units over 8 weeks and have not responded to, lost response to, or ineligible for erythropoiesis-stimulating agents.

The approval of the first-in-class oligonucleotide telomerase inhibitor was based on results of the randomized, double-blind, placebo-controlled, multicenter IMerge trial. The study included 178 patients with MDS who received imetelstat 7.1 mg/kg intravenously or placebo during 28-day treatment cycles until disease progression or unacceptable toxicity.

The ≥8-week red blood cell transfusion independence rate was 39.8% in the imetelstat group versus 15% in the placebo group (P<.001). The ≥24-week red blood cell transfusion independence rate was 28% and 3.3%, respectively (P<.001).

The most common side effects were laboratory abnormalities, decreased platelets, decreased white blood cells, decreased neutrophils, increased aspartate aminotransferase, increased alkaline phosphatase, increased alanine aminotransferase, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.

Reference

FDA approves imetelstat for low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia. US Food and Drug Administration. Published June 6, 2024. Accessed September 4, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-imetelstat-low-intermediate-1-risk-myelodysplastic-syndromes-transfusion-dependent