Imetelstat Receives FDA Advisory Committee Support for Anemia in Lower-Risk MDS

During a meeting on Thursday, March 14, the US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) voted that the benefits of imetelstat for managing anemia in lower-risk myelodysplastic syndromes (MDS) outweigh the risks with a count of 12 in favor and two against.

Despite the supportive vote, many of the panel members called for clearer understanding of the subset of patients who are likely to benefit.

Michael Sekeres, MD, of Sylvester Comprehensive Cancer Center, a previous chair for the ODAC who voted in favor of imetelstat, said, “I believe the benefits associated with the drug justify the risks. The benefits are real. When you have 40% of the patients who previously depended on red blood cell [RBC] transfusions who no longer need transfusions, and this outcome lasts for an average of one year, I think that’s a significant benefit.”

Jacqueline Garcia, MD, of Dana-Farber Cancer Institute, who also voted in favor of imetelstat, said, “I appreciate the access to details from both FDA and company perspectives and the raw data beyond the published paper. The stories from the patients were extremely meaningful and impactful and really mimic what I hear from my own patients for what they would like and what they want and what’s important to them . . . I look forward to the correlates and biomarker data that might come out in the future.”

Included in the “no” vote, was committee chair, Ravi Madan, MD, of the Center for Cancer Research of the National Cancer Institute.

Imetelstat for Anemia Treatment Supported by ODAC

The New Drug Application for imetelstat, a first-in-class telomerase inhibitor developed by Geron Corporation, seeks an indication as an injectable therapy for transfusion-dependent anemia in adult patients with lower-risk MDS who are not eligible for erythropoiesis-stimulating agents.

The application was supported by findings from the phase III IMerge trial published in The Lancet. The IMerge trial reported that imetelstat achieved significantly higher rates of RBC transfusion independence for at least eight consecutive weeks compared with placebo (P<.001).

Those who achieved transfusion independence had a median duration of transfusion independence approaching one year. Significant efficacy outcomes were observed across MDS subgroups regardless of ring sideroblast status.

“Based on the highly differentiated qualities of imetelstat reported in this study, we believe that, if approved by regulatory authorities, imetelstat could substantially improve the treatment paradigm in certain patients with lower-risk MDS,” Faye Feller, MD, Executive Vice President and Chief Medical Officer of Geron, previously commented.

Additionally, the Marketing Authorization Application for imetelstat has been validated by the European Medicines Agency and is undergoing regulatory review by the Committee for Medicinal Products for Human Use.

Following this vote, ODAC will decide on whether to provide a recommendation for the approval of imetelstat to the FDA. While a positive recommendation from the ODAC is nonbinding, the FDA typically follows advisory committee recommendations when making regulatory decisions.

Related: Panel Discusses New Data on Emerging Therapies in MDS: Imetelstat, KER-050, Plus More