The European Commission (EC) has approved the marketing authorization for imetelstat (RYTELO), a first-in-class telomerase inhibitor, as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to lower-risk myelodysplastic syndromes (MDS) in patients without a non-del 5q abnormality who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. This approval was announced on March 11, 2025.
The approval is based on the phase 3 IMerge clinical trial, which demonstrated the therapy reduced the need for red blood cell transfusions for enrolled patients in the first 24 weeks of treatment compared with placebo, according to the press release from Geron Corporation, the manufacturer of the therapy.
“I am thrilled that the European Commission has approved RYTELO in LR-MDS. The long-term and durable responses observed in the phase 3 IMerge study reinforce the practice-changing potential of telomerase inhibition as a clinically meaningful and differentiated option for the treatment of lower-risk MDS,” said Uwe Platzbecker, MD, an investigator on the IMerge trial and CMO at the University Hospital Carl Gustav Carus Dresden in Germany.“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.”
