FDA Approves New Treatment for Patients With MDS and Transfusion-Dependent Anemia

The Food and Drug Administration approved imetelstat (Rytelo, Geron Corporation), a first-in-class oligonucleotide telomerase inhibitor, to treat certain patients with lower-risk myelodysplastic syndromes with transfusion-dependent anemia.

Imetelstat works by inhibiting telomerase enzymatic activity. It is indicated for patients with low-to-intermediate-1 risk MDS who require four or more red blood cell units over eight weeks who have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents.

FDA based its approval of imetelstat on results from the IMerge Phase 3 clinical trial, which found that treatment with imetelstat led to higher rates of red blood cell transfusion independence versus placebo for at least eight consecutive weeks (39.8% versus 15%, respectively) and at least 24 weeks (28% versus 3.3%, respectively). Furthermore, the study demonstrated that transfusion independence was durable and sustained, with a median duration for 8-week responders and 24-week responders of approximately 1 year and 1.5 years, respectively.

Full prescribing information is available online.