The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending the approval of Rytelo (imetelstat) for the treatment of adult patients with transfusion-dependent (TD) anemia due to very low, low, or intermediate-risk myelodysplastic syndromes (LR-MDS) without an isolated deletion 5q cytogenetic abnormality. These are patients who have had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.
The European Commission (EC) is expected to make a final decision on the marketing authorization application in the coming months. If approved, Rytelo would be the first and only telomerase inhibitor available in Europe.
Clinical Trial Data
The CHMP's positive opinion was based on results from the IMerge Phase 3 clinical trial. The trial assessed the benefit of Rytelo in patients with transfusion-dependent anemia due to very low, low, or intermediate-risk MDS. Results showed a reduction in the need for red blood cell transfusions in the first 24 weeks of treatment compared to placebo, as observed in the double-blind controlled study.
Safety Profile
The most common side effects observed in the clinical trial were thrombocytopenia, leukopenia, neutropenia, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), increased alkaline phosphatase (ALP), asthenia, and headache. These side effects will be closely monitored if the drug is approved for use.
Dosing and Administration
If approved by the EC, Rytelo will be available as 47 mg and 188 mg lyophilized powder for reconstitution in a solution that is administered as an intravenous infusion. Treatment with Rytelo should be administered and monitored under the supervision of physicians and healthcare professionals experienced in hematologic diseases and their treatment.
Management Perspective
"The positive CHMP opinion is an important step towards our goal to optimize value and patient access to RYTELO in the European Union, where we look forward to the opportunity to make this important new treatment option for LR-MDS patients available in select markets," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. "We are deeply appreciative to the patients, caregivers, advocates, and investigators across the EU who contributed to the clinical development of RYTELO."
