The US Food and Drug Administration (FDA) has approved Geron’s Rytelo (imetelstat) to treat adults with lower-risk myelodysplastic syndromes (MDS) who have transfusion-dependent anaemia.
Patients eligible for the oligonucleotide telomerase inhibitor will have low- to intermediate-1 risk MDS, have anaemia requiring four or more red blood cell units over eight weeks, and will have not responded to, lost response to, or cannot be treated with erythropoiesis-stimulating agents.
Lower-risk MDS is a group of blood cancers that often progress to require increasingly intensified management of key symptoms such as anaemia.
Symptomatic patients frequently become red blood cell transfusion dependent, which has been associated with short- and long-term clinical consequences that reduce quality of life and shorten life expectancy.
The FDA’s decision on Rytelo, administered as an intravenous infusion every four weeks, was supported by positive results from the late-stage IMerge trial, in which the therapy demonstrated significantly higher rates of red blood cell transfusion independence (RBC-TI) versus placebo for eight consecutive weeks or longer.
RBC-TI was durable and sustained in the Rytelo-treated cohort, with a median RBC-TI duration for eight-week responders and 24-week responders of approximately one year and 1.5 years, respectively.
An exploratory analysis also showed improvements in haemoglobin levels for Rytelo versus placebo, Geron said, adding that clinically meaningful efficacy results from the study were observed across key MDS subgroups irrespective of ring sideroblast status, baseline transfusion burden and IPSS risk category.
IMerge investigator, Rami Komrokji, Moffitt Cancer Center, said: “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 practise 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.”
Also commenting on the FDA’s decision, John Scarlett, Geron’s chairman and chief executive officer, said: “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.”