EMA Validates Imetelstat Marketing Authorization Application for Lower-Risk MDS Treatment

The European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for imetelstat, a first-in-class investigational telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndromes (MDS).

The application was submitted in early September 2023 and is now under regulatory review by the European Committee for Medicinal Products for Human Use. The review applies to all 27 EU member states, Iceland, Norway, and Liechtenstein. It is expected to take approximately 14 months.

The MAA is based on results from IMerge phase III trial, which was a randomized, double-blind, placebo-controlled study evaluating imetelstat in patients with heavily transfusion dependent non-del(5q) lower-risk MDS who relapsed or were refractory to erythropoiesis stimulating agents.

The trial showed the eight-week transfusion independence rate was significantly higher with imetelstat than with placebo (P<0.001). The median duration of transfusion dependence approached one year for patients who received imetelstat and showed a transfusion independence response at eight weeks. The trial also showed a significant increase in mean hemoglobin levels in patients who received imetelstat.

Results from the phase III IMerge trial were presented by Amer Zeidan, MBBS, MHS, and colleagues during the 2023 American Society of Clinical Oncology Annual Meeting and by Uwe Platzbecker, MD, and colleagues during the 2023 European Hematology Association Congress.

Source: Business Wire, September 2023