FDA says ‘not clear’ magnitude, durability of RBC-TI outweighs Geron drug risks

In background information prepared by the Food and Drug Administration for the panel members of the Oncology Drugs Advisory Committee Meeting being convened on March 14 to discuss whether the efficacy of Geron’s imetelstat demonstrated on Study 63935937MDS3001 in adult patients with lower-risk myelodysplastic syndromes, or MDS, outweigh the risks of treatment, the FDA staff stated: “FDA acknowledges that the results of Study MDS3001-Phase 3 met the statistical goals for the primary endpoint of 8-week RBC-TI and key secondary endpoint of 24-week RBC-TI, but the following issues in the study design and efficacy results were identified: It is not clear that the magnitude and durability of RBC-TI outweighs the risks of treatment with imetelstat for patients with lower-risk MDS; The HI-E, CR, PR, and OS results are not supportive of a disease-modifying treatment effect; The patient-reported outcomes are not supportive of a treatment effect.”