European Commission approves Rytelo for LR- MDS-related anaemia

The European Commission (EC) has granted the first telomerase inhibitor approval in the EU for adults with transfusion-dependent anaemia due to very low, low, or intermediate-risk myelodysplastic syndromes (LR-MDS). The marketing authorisation for Rytelo ^® (imetelstat) applies to those without an isolated deletion 5q abnormality who are ineligible for or unresponsive to erythropoiesis-stimulating agents (ESAs). It marks a significant milestone for patients with LR- MDS-related anaemia who have limited treatment options beyond ESAs.

This decision for Rytelo is applicable across all 27 EU Member States, as well as Iceland, Norway, and Liechtenstein. This new approval by the EC follows the US FDA’s approval in June 2024.

Evidence from clinical trials for Rytelo in LR- MDS-related anaemia

The marketing authorisation is supported by data from the IMerge Phase III trial, which demonstrated that Rytelo significantly reduced red blood cell transfusion dependency in LR-MDS patients. The study found that a substantial proportion of patients achieved prolonged transfusion independence, with some maintaining responses for over a year. The drug, administered intravenously every four weeks, inhibits telomerase activity, a key factor in malignant stem cell proliferation in bone marrow.

The therapy’s overall benefit-risk profile was deemed favourable for patients with limited alternatives after ESA failure. Therefore, Rytelo’s approval marks a significant advancement for patients seeking long-term transfusion independence.

“As the first and only treatment of its kind, Rytelo represents an important new option – significantly reducing the need for red blood cell transfusions for people living with LR-MDS who are battling debilitating symptoms like anaemia and fatigue,” Dr Joseph Eid, Executive Vice President of Research and Development at Geron stated.

“The long-term and durable responses observed in the Phase III IMerge study reinforce the practice-changing potential of telomerase inhibition as a clinically meaningful and differentiated option for the treatment of lower-risk MDS,” Dr Uwe Platzbecker, co-lead author of the Phase III trial added.

Market access and future plans

Geron plans to launch Rytelo (imetelstat) in select European countries by 2026, pending national reimbursement approvals. Additionally, the EMA has recommended maintaining Rytelo’s orphan drug designation, potentially securing ten years of market exclusivity.