FDA Approves Rytelo for Lower-Risk Myelodysplastic Syndrome

The US Food and Drug Administration (FDA) has recently given the green light to Geron’s Rytelo (imetelstat) for treating adults suffering from lower-risk myelodysplastic syndromes (MDS) who are dependent on blood transfusions due to anaemia. This new therapy is aimed at patients with low to intermediate-1 risk MDS who need at least four units of red blood cells over an eight-week period and who have not responded, no longer respond, or cannot be treated effectively with erythropoiesis-stimulating agents.

Myelodysplastic syndromes are a spectrum of blood cancers that can escalate, necessitating more intensive management of symptoms like anaemia. For patients with lower-risk MDS, becoming dependent on red blood cell transfusions is a common progression. This dependency is linked to both short-term and long-term health consequences, which can significantly diminish quality of life and reduce overall life expectancy.

The approval of Rytelo by the FDA is based on encouraging outcomes from the late-stage IMerge clinical trial. Administered via intravenous infusion every four weeks, Rytelo demonstrated markedly higher rates of red blood cell transfusion independence (RBC-TI) compared to a placebo, lasting for eight consecutive weeks or more. In the group treated with Rytelo, RBC-TI was both durable and sustained, with eight-week responders and 24-week responders experiencing a median RBC-TI duration of roughly one year and 1.5 years, respectively.

The IMerge trial also included an exploratory analysis revealing that Rytelo improved haemoglobin levels more effectively than the placebo. Geron noted that significant efficacy results were seen across various MDS subgroups, regardless of ring sideroblast status, baseline transfusion burden, and IPSS risk category.

Dr. Rami Komrokji of the Moffitt Cancer Center, an investigator in the IMerge trial, emphasized the significance of this approval. He pointed out that for patients with lower-risk MDS and transfusion-dependent anaemia, current treatment options are limited, often leading patients to cycle through available therapies. The approval of Rytelo could thus be a game-changer in clinical practice. Dr. Komrokji stated, "For patients with lower-risk MDS and anaemia who are transfusion dependent, we have very few options today and often cycle through available therapies, making the approval of Rytelo potentially practice changing for us… The treatment goal for patients with lower-risk MDS and anaemia is transfusion-independence and before today, this wasn’t possible for many patients."

John Scarlett, Geron’s chairman and chief executive officer, also commented on the FDA’s decision, noting the potential benefits for eligible patients. He remarked, "With the approval and availability of Rytelo, we believe eligible patients with lower-risk MDS can potentially experience meaningful clinical benefit, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anaemia."

This new treatment option is expected to provide a significant improvement in the management of lower-risk MDS, offering new hope for patients who previously had limited therapeutic choices.