The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).
Imetelstat is an investigational first-in-class telomerase inhibitor designed to block the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies. The application is supported by data from the IMerge study (ClinicalTrials.gov Identifier: NCT02598661), which evaluated the efficacy and safety of imetelstat in patients with International Prognostic Scoring System (IPSS) low or intermediate-1 risk MDS. In the phase 3 portion, 178 participants were randomly assigned 2:1 to receive either imetelstat via intravenous injection every 4 weeks or placebo.
The primary endpoint was the rate of red blood cell transfusion independence (RBC-TI) lasting at least 8 weeks, defined as the proportion of patients without any RBC transfusion for at least 8 consecutive weeks since entry to the trial.
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Results showed that a significantly higher proportion of patients treated with imetelstat met the primary endpoint of 8-week transfusion independence vs placebo (P <.001), with median TI duration approaching 1 year among 8-week responders. Imetelstat-treated patients also achieved significant increases in mean hemoglobin levels over time compared with placebo (P <.001). Improvements were noted across key MDS subgroups, regardless of ring sideroblast status, baseline transfusion burden and IPSS risk category.
“The FDA’s acceptance of our New Drug Application is an important landmark along our steadfast journey to bring telomerase inhibition with imetelstat to the market,” said John A. Scarlett, MD, Geron’s Chairman and Chief Executive Officer. “We look forward to continuing our collaboration with the FDA toward the goal of bringing imetelstat to the many patients for whom we believe this treatment could make a significant difference.”
A Prescription Drug User Fee Act target date of June 16, 2024 has been assigned to the application.
This article originally appeared on MPR
