FDA Approves Imetelstat for Patients With Lower-Risk MDS With Transfusion-Dependent Anemia

The US Food and Drug Administration (FDA) has approved imetelstat for adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia, according to a press release from Geron, the manufacturer of the first-in-class telomerase inhibitor.

Imetelstat is indicated for patients who require four or more red blood cell units over eight weeks and have not responded to, lost response to, or are ineligible for erythropoiesis-stimulating agents.

The approval was based on results of the randomized, double-blind, placebo-controlled, multicenter IMerge study. For eight weeks, intravenous imetelstat demonstrated higher rates of red blood cell transfusion-independence compared with placebo (39.8% [95% CI, 30.9–49.3] vs 15.0% [95% CI, 7.1–26.6], respectively; P < 0.001).

The most common grade 3 or 4 adverse events were neutropenia (72%) and thrombocytopenia (65%), which resolved to grade 2 or lower in under four weeks for more than 80% of patients. Cytopenias were manageable with dose modifications.

“For patients with lower-risk MDS and anemia who are transfusion-dependent, we have very few options today and often cycle through available therapies, making the approval of [imetelstat] potentially practice-changing,” said Rami Komrokji, MD, Vice Chair of the Malignant Hematology Department at the Moffitt Cancer Center and an investigator of the IMerge trial, in the press release. “The treatment goal for patients with [lower-risk] MDS and anemia is transfusion-independence. Before today, this wasn’t possible for many patients.”