The drug showed statistically significant and clinically meaningful efficacy results across key subgroups.
The FDA has accepted the filing of a New Drug Application (NDA) for imetelstat (Genron Corp), a telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndrome (MDS), according to a statement released by the company.
“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, chairman and CEO of Geron, in a press release. “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.”
The submission is based on results from phase 3 of the IMerge double-blind, randomized clinical trial evaluating imetelstat in patients with IPSS Low or Intermediate-1 risk (lower risk) transfusion dependent MDS who relapsed after, were refractory to, or were ineligible for erythropoiesis stimulating agent (ESA) treatment, had not received prior treatment with either a HMA or lenalidomide, and were non-deletion 5q.
The primary efficacy endpoint of the trial is the rate of red blood cell transfusion independence (RBC-TI) lasting at least 8 weeks, which was defined as the proportion of patients without any RBC transfusion for at least 8 consecutive weeks since entry to the trial. Key secondary endpoints were the rate of RBC-TI lasting at least 24 weeks, the duration of transfusion independence, and the rate of hematologic improvement erythroid.
The primary endpoint of 8-week transfusion independence was significantly higher with imetelstat versus placebo (p < 0.001), with median transfusion independence duration approaching 1 year for imetelstat 8-week transfusion independence responders. Additionally, mean hemoglobin levels in imetelstat-treated patients increased significantly (p < 0.001) over time compared to placebo patients.
“FDA acceptance of our NDA is a significant milestone for both Geron and the MDS community, as there remain few treatment options and significant unmet needs, particularly for patients with difficult-to-treat subtypes of this cancer,” said Faye Feller, MD, executive vice president and chief medical officer at Geron, in the press release. “We believe that the IMerge Phase 3 data reflect the truly unique attributes of imetelstat, and, if approved, we expect imetelstat will change the standard of care in lower risk MDS.”
BusinessWire. Geron announces FDA acceptance of new drug application for imetelstat for the treatment of lower risk MDS. News release. Accessed on August 22, 2023. Published on August 21, 2023. Accessible at https://www.businesswire.com/news/home/20230821960985/en.