FDA Approves RYTELO™ for Treating Lower-Risk MDS with Transfusion-Dependent Anemia

Geron Corporation has announced that the U.S. Food and Drug Administration (FDA) has approved RYTELO™ (imetelstat) for the treatment of adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have transfusion-dependent anemia. This approval covers patients who require four or more red blood cell units over eight weeks and have not responded to or are ineligible for erythropoiesis-stimulating agents (ESAs).

RYTELO marks a significant development as the first telomerase inhibitor approved by the FDA, offering new hope for patients with lower-risk MDS. This is a progressive blood cancer where many patients become dependent on red blood cell transfusions, severely impacting their quality of life and carrying long-term clinical consequences. 

John A. Scarlett, M.D., Geron's Chairman and CEO, emphasized the potential clinical benefits for patients, noting that RYTELO could offer more than 24 weeks of relief from the burden of transfusions and symptomatic anemia. He expressed gratitude to everyone involved in the development and approval process, including patients, families, clinicians, and Geron employees.

Lower-risk myelodysplastic syndromes (LR-MDS) is a condition that often necessitates intensive management of anemia and fatigue. These patients frequently need red blood cell transfusions, which can lead to both short- and long-term health issues and reduce survival rates. Given the limited treatment options for patients who do not respond to ESAs, there is a significant need for therapies that can provide extended transfusion independence.

The FDA approval for RYTELO was based on results from the IMerge Phase 3 clinical trial, which was published in The Lancet. The trial demonstrated that RYTELO significantly improved rates of red blood cell transfusion independence (RBC-TI) compared to placebo. Specifically, 39.8% of patients on RYTELO achieved RBC-TI for at least eight weeks versus 15.0% on placebo. For a duration of at least 24 weeks, 28.0% of patients on RYTELO achieved RBC-TI compared to just 3.3% on placebo. The median RBC-TI duration was about one year for eight-week responders and 1.5 years for 24-week responders.

Additionally, in an exploratory analysis, median increases in hemoglobin were 3.6 g/dL for RYTELO-treated patients compared to 0.8 g/dL for placebo. These efficacy results were consistent across key MDS subgroups, regardless of ring sideroblast (RS) status or baseline transfusion burden.

The safety profile of RYTELO was generally well-characterized, with manageable and short-lived cytopenias such as thrombocytopenia and neutropenia. The most common Grade 3/4 adverse reactions were neutropenia (72%) and thrombocytopenia (65%), which typically lasted less than two weeks. These cytopenias were usually manageable with dose adjustments.

Adverse reactions occurring in 10% or more of patients included decreased platelets, white blood cell counts, increased liver enzymes, fatigue, prolonged partial thromboplastin time, muscle or joint pain, COVID-19 infections, and headaches. Serious adverse reactions such as sepsis and gastrointestinal hemorrhage were less common.

RYTELO is administered intravenously over two hours every four weeks. It works by inhibiting the telomerase enzyme, which allows cancer cells to divide uncontrollably. Geron is committed to ensuring broad access to RYTELO through its REACH4RYTELO™ Patient Support Program, which provides resources to support access and affordability for eligible patients.

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