Imetelstat Approved in European Union for Anemia, Lower-Risk MDS

The European Commission (EC) has approved imetelstat for adult patients with transfusion-dependent anemia due to lower-risk myelodysplastic syndromes (LR-MDS), according to a press release from Geron Corporation, the developer of the drug.¹

The drug is indicated for patients without an isolated deletion 5q cytogenetic (non-del 5q) abnormality who had an unsatisfactory response to or are ineligible for erythropoiesis-stimulating agents.¹

Imetelstat was previously approved in the United States in June 2024. Both approvals were based on results of the phase 3, double-blind, placebo-controlled IMerge study, which showed disease-modifying activity and durable transfusion independence of about 1 year with imetelstat. The rate of transfusion independence was 40% in the imetelstat group versus 15% in the placebo group (P=0.0008).²

Ninety-one percent of patients receiving imetelstat and 47% receiving placebo experienced grade 3-4 treatment-emergent adverse events, most commonly neutropenia (68% with imetelstat vs 3% with placebo) and thrombocytopenia (62% vs 8%). No treatment-related deaths were reported.²

“I am thrilled that the European Commission has approved RYTELO [imetelstat] in LR-MDS,” Uwe Platzbecker, MD, co–lead author of the IMerge trial and chief medical officer at the University Hospital Carl Gustav Carus Dresden in Germany, said in the press release. “Physicians and patients in Europe are now one step closer to accessing a novel treatment that, in addition to having a generally manageable safety profile, has the potential to provide extended and continuous red blood cell transfusion independence.”

References

1. Geron announces European Commission approval of RYTELO® (imetelstat), a first-in-class telomerase inhibitor, for the treatment of adults with transfusion-dependent anemia due to lower-risk MDS. News release. BusinessWire. March 11, 2025. Accessed March 13, 2025. https://www.businesswire.com/news/home/20250311485595/en/Geron-Announces-European-Commission-Approval-of-RYTELO%C2%AE-imetelstat-a-First-in-Class-Telomerase-Inhibitor-for-the-Treatment-of-Adults-With-Transfusion-Dependent-Anemia-Due-to-Lower-Risk-MDS
2. Platzbecker U, Santini V, Fenaux P, et al. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024;403(10423):249-260. doi:10.1016/S0140-6736(23)01724-5