FDA Approves Geron's RYTELO for MDS-Related Anemia

The US Food and Drug Administration (FDA) has approved Geron's RYTELO (imetelstat) for adults diagnosed with low to intermediate-1 risk myelodysplastic syndromes (MDS) and who suffer from transfusion-dependent anemia. This approval represents a crucial advancement for patients needing frequent red blood cell transfusions, specifically those requiring four or more transfusions over an eight-week period. These patients are either unresponsive to, have lost responsiveness to, or are not candidates for erythropoiesis-stimulating agents.

This FDA decision is largely based on the positive outcomes from the IMerge Phase III clinical trial. The trial demonstrated that RYTELO significantly increased the duration of red blood cell transfusion independence in comparison to a placebo. Both the primary and key secondary endpoints of the trial were met, highlighting RYTELO's potential to provide durable and sustained responses for patients with MDS.

The safety profile of RYTELO was well-documented during the trial, with manageable cytopenias being the most common adverse reactions. Specifically, neutropenia and thrombocytopenia were the most frequent Grade 3/4 side effects, but these typically resolved within a short timeframe.

Upon announcing the approval, Geron's chairman and CEO, John Scarlett, expressed optimism about the potential clinical benefits for eligible patients with lower-risk MDS. He emphasized that RYTELO could offer patients more than 24 weeks of relief from the burden of red blood cell transfusions and symptomatic anemia. Scarlett also highlighted that the approval of RYTELO as the first telomerase inhibitor underscores the effectiveness of Geron’s scientific research and the dedication of its team. He extended gratitude to the patients, their families, advocates, clinicians, study coordinators, site personnel, scientists, and both current and former Geron employees and collaborators, all of whom played essential roles in achieving this milestone.

In March 2024, FDA advisers supported the approval of imetelstat, stating that the benefits outweigh the associated risks, based on data from the Phase III IMerge trial in MDS patients.

This approval is a significant development for the field of blood cancer treatment, specifically for patients with myelodysplastic syndromes who often face a challenging prognosis. RYTELO's approval offers a new avenue for treatment, potentially improving the quality of life for many patients and reducing their dependence on frequent blood transfusions.