Geron’s Rytelo Receives FDA Approval for Lower-Risk Myelodysplastic Syndromes Treatment

1. FDA Approval: After 33 years, Geron Corporation's Rytelo (imetelstat) has received FDA approval for the treatment of adult patients with lower-risk myelodysplastic syndromes (MDS) who have transfusion-dependent anemia and have not responded to or are ineligible for erythropoiesis-stimulating agents (ESA).
2. Indication: Rytelo is indicated for patients with low-to-intermediate-1 risk MDS who require four or more red blood cell units over eight weeks and have not responded to or are ineligible for ESA.
3. Mechanism of Action: Rytelo is a telomerase inhibitor that works by inhibiting telomerase enzymatic activity, which helps to control unregulated cell division in MDS.
4. Clinical Trial Results: The IMerge Phase III clinical trial demonstrated significantly higher rates of red blood cell transfusion independence (RBC-TI) with Rytelo compared to a placebo, in periods of both eight weeks consecutively and a minimum of 24 weeks.
5. Potential Benefits: The approval of Rytelo has the potential to help patients reduce the need for red blood cell transfusions and alleviate symptomatic anemia, providing extended periods of transfusion independence.
6. Patient Support Program: Geron's REACH4RYTELO Patient Support Program offers resources to support access and affordability for eligible patients prescribed Rytelo.
7. Safety Profile: Rytelo's safety profile includes the risk of thrombocytopenia, with new or worsening Grade 3 or 4 decreased platelets occurring in 65% of patients with MDS treated with Rytelo in the clinical trial.