Geron Announces FDA Approval of RYTELO? for Lower-Risk MDS

SciFocus/June 24, 2024 -- Geron Corporation (Nasdaq: GERN), a leading biopharmaceutical company, has announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for RYTELO? (imetelstat), the first-in-class telomerase inhibitor, for the treatment of adult patients with lower-risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia. This approval covers patients who are ineligible or have relapsed after erythropoiesis-stimulating agents (ESA) treatment.

Key Highlights:

- First-in-Class Treatment: RYTELO? is the first telomerase inhibitor approved for MDS, offering a new option for patients with transfusion-dependent anemia.

- Clinical Trial Success: The approval is based on the Phase III IMerge clinical trial, which showed significant and sustained transfusion independence and hemoglobin level improvements.

- Addressing High Unmet Need: Lower-risk MDS is a progressive blood cancer with limited treatment options for ESA-ineligible patients, making RYTELO? a significant advancement.

- Safety Profile: Common adverse reactions include manageable thrombocytopenia and neutropenia, with most side effects resolving within four weeks.

Approval Details:

- Indication: RYTELO? is approved for adult patients with low- to intermediate-1 risk MDS with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks, who have not responded to or lost response to ESAs.

- Clinical Benefits: The IMerge trial demonstrated that RYTELO? significantly increased rates of red blood cell transfusion independence, with durable effects observed for up to 1.5 years in some patients.

- Safety and Adverse Reactions: The most common Grade 3/4 adverse reactions were neutropenia (72%) and thrombocytopenia (65%). These were generally short-lived and manageable with dose adjustments.

Expert Opinions:

- Dr. John A. Scarlett, CEO of Geron: "With the approval and availability of RYTELO, eligible patients with lower-risk MDS can potentially experience meaningful clinical benefits, especially freedom from the burden of red blood cell transfusions and symptomatic anemia."

- Dr. Rami Komrokji, Moffitt Cancer Center: "RYTELO's approval is practice-changing, providing a new treatment option that offers sustained transfusion independence and hemoglobin improvements across key MDS subgroups."

Impact and Future Outlook:

- Patient Advocacy: Carol Roberts of PBCers emphasizes the importance of new treatment options for improving quality of life for PBC patients, who often feel dismissed and uncertain about their disease progression.

- European Submissions: RYTELO? has been submitted for approval to the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA), with decisions expected in late 2024.

RYTELO? represents a significant advancement in the treatment of lower-risk MDS, addressing a critical unmet need and offering new hope to patients with transfusion-dependent anemia. Geron's commitment to innovation in blood cancer therapy is poised to make a profound impact on patient care.

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