Geron Corporation (Nasdaq: GERN), a biopharmaceutical company focused on blood cancer treatments, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the approval of RYTELO™ (imetelstat) for the treatment of adult patients with transfusion-dependent anemia due to very low, low, or intermediate-risk myelodysplastic syndromes (LR-MDS). This recommendation applies to patients who lack an isolated deletion 5q cytogenetic abnormality and have had an unsatisfactory response to, or are ineligible for, erythropoietin-based therapy. The European Commission (EC) will now review the CHMP’s recommendation and is expected to make a final decision on marketing authorization in the coming months.
Geron’s CEO, John A. Scarlett, M.D., expressed optimism about the CHMP opinion, highlighting its significance for improving patient access to RYTELO in the European Union. If approved, RYTELO would become the first telomerase inhibitor available in Europe for the treatment of LR-MDS patients. Dr. Scarlett also thanked the patients, caregivers, and healthcare professionals who contributed to the clinical development of RYTELO.
The CHMP’s review was based on data from the IMerge Phase 3 clinical trial, which demonstrated that RYTELO significantly reduced the need for red blood cell transfusions in the first 24 weeks compared to a placebo. The most common side effects observed were thrombocytopenia, leukopenia, neutropenia, increased liver enzymes (AST, ALT), increased alkaline phosphatase, as well as asthenia and headache.
RYTELO is administered intravenously and, if approved, will be available in two dosages: 47 mg and 188 mg lyophilized powder for reconstitution. Treatment should be managed by healthcare professionals experienced in hematologic diseases.
LR-MDS is a blood cancer where abnormal bone marrow cells often cause progressive anemia and fatigue, leading many patients to become transfusion-dependent. Current treatment options for transfusion-dependent LR-MDS patients who fail erythropoiesis-stimulating agents (ESAs) are limited, creating a need for alternative therapies like RYTELO that offer potential for sustained red blood cell transfusion independence.
RYTELO is a first-in-class telomerase inhibitor that works by inhibiting telomerase enzymatic activity, which is crucial in the uncontrolled cell division observed in LR-MDS. Developed and owned by Geron, RYTELO is currently FDA-approved for adult patients with low-to-intermediate-risk LR-MDS and transfusion-dependent anemia who are unresponsive or ineligible for ESAs.
Regarding safety, RYTELO may cause thrombocytopenia and neutropenia, and patients must be monitored for infections, bleeding, and other side effects. Infusion-related reactions, including headache and hypertension, are possible and can be managed with premedication and monitoring during the infusion.
If approved by the EC, RYTELO will provide a critical new treatment option for patients suffering from LR-MDS, a condition with significant unmet medical need. The approval would follow the FDA’s earlier approval of RYTELO for similar indications in the U.S.
In addition to the European review, RYTELO’s marketing authorization is under review by other regulatory authorities, including in Canada, Australia, Singapore, Switzerland, and South Korea.
