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On Thursday, the FDA approved Geron Corporation's GERN Rytelo (imetelstat) for adult patients with low-to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia . The approval covers patients requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents. In March, by a 12-to-2 margin, the FDA adcomm panel voted in favor of the benefits of imetelstat, stating that the associated risks and toxicities of the treatment appear to be manageable .
The FDA approval is based on IMerge Phase 3 trial results. The IMerge trial met its primary and key secondary endpoints, with Rytelo demonstrating significantly higher rates of red blood cell transfusion independence (RBC-TI) versus placebo for at least eight consecutive weeks (Rytelo 39.8%; placebo 15.
0%) and at least 24 weeks (Rytelo 28.0%; placebo 3.3%.
RBC-TI was durable and sustained in the Rytelo-treated population, with a median RBC-TI duration for 8-week responders and 24-week responders of approximately one year and 1.5 years, respectively. In an exploratory analysis of Rytelo-treated patients achieving ≥8-week RBC-TI, median increases in hemoglobin were 3.
6 g/dL for Rytelo and 0.8 g/dL for placebo. Clinically meaningful efficacy results were observed across key MDS subgroups irrespective of ring sideroblast status, baseline transfusion burden, and IPSS risk category.
Imet.