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Evaluating Imetelstat’s Efficacy and Safety: A Comprehensive Insight into its Impact on MDS Treatment

The recent meeting of the FDA’s Oncologic Drugs Advisory Committee (ODAC) has brought imetelstat, a promising treatment for myelodysplastic syndrome (MDS), into the limelight. With a significant focus on the drug’s risk-benefit balance, the committee’s deliberations shed light on imetelstat’s potential as a second-line treatment for low- to intermediate-risk MDS patients, reflecting a pivotal moment in the ongoing quest to enhance therapeutic options for this patient demographic.

Key Points:

• The ODAC voted 12–2 in favor of imetelstat for low- to intermediate-risk MDS patients who are transfusion-dependent and unresponsive to or ineligible for erythropoiesis-stimulating agents (ESAs).
• Despite acknowledging the drug’s side effects, ODAC members believe these are manageable and that imetelstat met its clinical trial endpoints, indicating an improvement in patient quality of life.
• Patient testimonials highlighted at the meeting emphasized the significant impact of transfusion dependence on their lives, reinforcing the need for additional treatment options.
• Imetelstat, a telomerase inhibitor, demonstrated in the MDS3001 phase 3 trial that 39.8% of treated patients achieved red blood cell transfusion independence after 8 weeks, a notable increase from the 15.0% in the placebo group.
• The drug also met a secondary endpoint of 24-week transfusion independence, with 28.0% of the imetelstat group achieving this milestone versus 3.3% in the placebo group.
• FDA officials expressed concerns about the clinical significance of the 8-week RBC-TI period and mentioned that a 16-week or longer period is typically considered meaningful.
• High rates of grade 3 and grade 4 adverse events were observed in the imetelstat group, with neutropenia and thrombocytopenia being the most common.
• ODAC discussions included the need for future studies to better define patient populations that could benefit most from imetelstat, suggesting a focus on biomarkers and clinical parameters.

“The stories from the patients are extremely meaningful and impactful and really mimic what I hear from my own patients for what they would like, what they want, and what’s important to them in their time.”
– ODAC member Jacqueline S. Garcia, MD

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