Geron Corporation (Nasdaq: GERN), a biopharmaceutical company dedicated to revolutionizing blood cancer treatment, has received FDA approval for RYTELO™ (imetelstat). This approval is specifically for treating adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have transfusion-dependent anemia requiring four or more red blood cell units over eight weeks. These patients have either not responded to, lost response to, or are ineligible for erythropoiesis-stimulating agents (ESA).
John A. Scarlett, M.D., Chairman, and Chief Executive Officer of Geron, expressed optimism about the approval, highlighting its potential to offer significant clinical benefits. He emphasized the possibility of more than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia for eligible patients. Scarlett credited the milestone to Geron’s scientific prowess and the dedication of its team, thanking all those involved, including patients, families, advocates, clinicians, and collaborators.
Myelodysplastic syndromes (MDS) is a blood cancer that often leads to intensified symptom management, particularly for anemia and resulting fatigue. Many patients become dependent on red blood cell transfusions, which can significantly impact quality of life and survival. There is a pressing need for effective treatments, especially for patients who have not responded to current therapies. RYTELO offers promise in providing extended and continuous red blood cell transfusion independence.
The FDA approval of RYTELO is based on promising results from the IMerge Phase 3 clinical trial. This trial demonstrated significantly higher rates of red blood cell transfusion independence with RYTELO compared to placebo, both for at least eight consecutive weeks and for at least 24 weeks. The durability of response was notable, with a median duration of approximately one year for 8-week responders and 1.5 years for 24-week responders.
RYTELO also showed clinically meaningful increases in hemoglobin levels across various MDS subgroups, regardless of certain baseline characteristics. The safety profile of RYTELO was well-characterized, with manageable cytopenias being the most common adverse reactions. Thrombocytopenia and neutropenia were the most frequent Grade 3/4 adverse reactions but were generally short-lived and manageable with dose modifications.
In light of this approval, a conference call with Geron management is scheduled to discuss the FDA approval and the launch of RYTELO. Geron aims to ensure broad access to RYTELO through its REACH4RYTELO™ Patient Support Program, providing resources to support access and affordability for eligible patients.
RYTELO, an oligonucleotide telomerase inhibitor, is administered as an intravenous infusion every four weeks. It works by inhibiting telomerase enzymatic activity, addressing abnormal cell division characteristic of LR-MDS. Geron’s commitment to innovation in blood cancer treatment is evident in RYTELO’s status as the first and only FDA-approved telomerase inhibitor.
While RYTELO offers promise, it’s essential to consider safety information. Thrombocytopenia, neutropenia, and infusion-related reactions are potential risks associated with RYTELO. Patients should be closely monitored, and appropriate measures should be taken to manage adverse reactions.
In conclusion, the FDA approval of RYTELO marks a significant advancement in the treatment of lower-risk myelodysplastic syndromes. Geron is committed to supporting patients and healthcare providers in accessing this innovative therapy, with the goal of improving outcomes and quality of life for those affected by LR-MDS.
