FDA Greenlights Geron's Novel Telomerase Inhibitor for Blood Disorder

Geron Corporation has announced that the U.S. Food and Drug Administration (FDA) has granted approval for Rytelo (imetelstat), a treatment for patients with lower- to intermediate-risk myelodysplastic syndromes (MDS). MDS encompasses a variety of blood cancers caused by the abnormal formation or malfunctioning of blood cells. Geron's announcement led to a significant surge in its stock, which increased by more than 30% in Friday morning trading.

Rytelo is designed for MDS patients who suffer from transfusion-dependent anemia and who either do not respond to or are unsuitable for erythropoiesis-stimulating agents (ESA), the standard treatment. This approval marks a significant milestone as Rytelo is not only a first-in-its-class drug but also Geron’s first approved medication in its 34-year history.

Dr. Rami Komrokji, Vice Chair of the Malignant Hematology Department at Moffitt Cancer Center and a researcher in the pivotal IMerge trial, remarked on the limited treatment options currently available for patients with lower-risk MDS and transfusion-dependent anemia. He suggested that the approval of Rytelo could be a transformative development for clinical practice.

MDS is characterized by the failure of blood cells in the bone marrow to mature into healthy cells. In cases categorized as lower-risk, the disease often necessitates intensive management of symptoms such as anemia and can eventually lead to serious issues like organ dysfunction and heart complications.

The Phase III IMerge trial provided compelling evidence for Rytelo's efficacy. Patients treated with Rytelo experienced significantly higher rates of red blood cell transfusion independence (RBC-TI) compared to those receiving a placebo. Specifically, 28% of patients in the treatment group achieved RBC-TI for at least 24 weeks, compared to just 3% in the placebo group. For these responders, the median duration of RBC-TI was 1.5 years.

Administered intravenously every four weeks, Rytelo's most common side effects were described by Geron as "manageable and short-lived." The most frequent serious adverse effects were neutropenia (72%) and thrombocytopenia (65%), with these conditions lasting a median of less than two weeks.

In a related development, Bristol Myers Squibb’s Reblozyl achieved a label expansion in August 2023, establishing it as a first-line treatment for anemia in low-risk MDS patients who had not previously received ESA treatment. Reblozyl works by promoting the production of red blood cells, thereby reducing the need for transfusions. In a Phase III trial, 58.5% of patients treated with Reblozyl attained at least 12 weeks of RBC-TI, compared to 31.2% of patients treated with ESAs.

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