FDA Advisory Committee Supports Benefit of Imetelstat for Anemia in Lower-Risk MDS

The US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) passed a vote on whether the benefits of treatment with imetelstat outweigh the risks with a count of 12 in favor and two against during an advisory meeting on Thursday, March 14.

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

In favor for imetelstat, Jacqueline Garcia, MD, at Dana-Farber Cancer Institute, 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.”

Michael Sekeres, MD, at Sylvester Comprehensive Cancer Center, who previously served as chair of the ODAC, 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 transfusions who no longer need transfusions, and this outcome lasts for an average of one year, I think that’s a significant benefit.”

The New Drug Application (NDA) 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 myelodysplastic syndromes (MDS) who are not eligible for erythropoiesis-stimulating agents. 

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

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 red blood cell 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 at Geron, previously commented. 

Additionally, the Marketing Authorization Application for imetelstat was validated by the European Medicines Agency and is undergoing regulatory review by the European 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