FDA Approves Geron's Rytelo for Lower-Risk MDS Anemia

FDA Approves Geron's Rytelo for Lower-Risk MDS Anemia

The US Food and Drug Administration (FDA) has approved Geron Corporation's Rytelo (imetelstat) for adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia. This approval is intended for patients requiring red blood cell transfusions four or more times over eight weeks who have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents. The decision was based on the results of the IMerge Phase 3 clinical trial, which demonstrated significantly higher rates of red blood cell transfusion independence (RBC-TI) with imetelstat compared to a placebo.

The IMerge trial showed that 39.8% of patients achieved RBC-TI for at least eight weeks with imetelstat, compared to 3.3% with placebo. Additionally, 28% of patients experienced RBC-TI for 24 weeks or more with imetelstat. The most common toxicities reported in 10% or more of patients included decreased platelet counts, decreased white blood cells, decreased neutrophil counts, and increased levels of aspartate aminotransferase, alkaline phosphatase, and alanine aminotransferase. John A. Scarlett, MD, chairman and CEO of Geron, highlighted that Rytelo's approval as the first telomerase inhibitor marks a significant milestone in the treatment of blood cancers.

Shares of Geron rose over 30% following the approval. The company has set wholesale prices at $9,884 for a 188 mg vial and $2,471 for a 47 mg vial of Rytelo. Geron also anticipates data from the Phase 3 IMpactMF trial in relapsed/refractory myelofibrosis (MF) in 2026 due to lower-than-expected enrollment and mortality rates.


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