Geron has received the European Commission (EC) marketing authorisation for Rytelo (imetelstat) as a monotherapy to treat transfusion-dependent anaemia due to lower-risk myelodysplastic syndromes (LR-MDS).
LR-MDS is a blood cancer that often necessitates intensified management of symptoms such as anaemia and fatigue.
Rytelo is a telomerase inhibitor that works by inhibiting telomerase enzymatic activity.
The drug is indicated for adult patients with non-del 5q LR-MDS who have not responded to or are ineligible for erythropoietin-based therapies.
The EC authorisation follows the European Medicines Agency’s Committee of Orphan Medicinal Products (CHMP) positive opinion on Rytelo’s orphan drug designation in the EU.
The approval applies to all 27 European Union member states, as well as Iceland, Liechtenstein, and Norway.
Geron research and development executive vice president Joseph Eid said: “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.
“This approval from the European Commission, just nine months following approval in the US, underscores the positive benefit for these patients demonstrated in our clinical trials and we look forward to making this innovative therapy accessible to eligible patients in Europe.”
The marketing authorisation is supported by data from the IMerge Phase 3 clinical trial, which demonstrated Rytelo’s significant clinical benefit.
The trial showed a reduction in the need for red blood cell transfusions in patients with LR-MDS during the first 24 weeks of treatment compared to placebo.
Rytelo showed a safety profile that was well-characterised, with manageable side effects such as thrombocytopenia and neutropenia.
The US-based biotechnology company is preparing to commercialise Rytelo in select EU countries starting in 2026, subject to country-specific reimbursement agreements.
The company is also exploring opportunities to provide Rytelo to eligible patients through Expanded Access Programmes, including Named Patient Programmes.
Phase 3 IMerge study investigator and co-lead author Uwe Platzbecker said: “I am thrilled that the European Commission has approved RYTELO in LR-MDS.
“The long-term and durable responses observed in the Phase 3 IMerge study reinforce the practice-changing potential of telomerase inhibition as a clinically meaningful and differentiated option for the treatment of lower-risk MDS.
“Physicians and patients in Europe are now one step closer to accessing a novel treatment that, in addition to having a generally manageable safety profile, has the potential to provide extended and continuous red blood cell transfusion independence.”
