FOSTER CITY, Calif. – Geron (NASDAQ:GERN) Corporation (NASDAQ:GERN), a biopharmaceutical company, has received a favorable vote from the U.S. Food and Drug Administration's (FDA) Oncologic Drugs Advisory Committee (ODAC) regarding its drug imetelstat.
The committee voted 12 to 2 in support of the clinical benefit/risk profile of imetelstat for the treatment of transfusion-dependent anemia in adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) who are not eligible for or have not responded to erythropoiesis-stimulating agents (ESAs).
The ODAC's positive opinion is based on data from the IMerge Phase 3 clinical trial, which demonstrated a significant increase in the rate of red blood cell transfusion independence lasting at least eight weeks for patients treated with imetelstat compared to placebo.
Moreover, the trial showed that 28% of patients treated with imetelstat achieved at least 24-week red blood cell transfusion independence, a key secondary endpoint of the study. The median duration of this transfusion independence was 80 weeks for those patients.
Despite the favorable committee vote, the FDA is not obligated to follow the ODAC's recommendation, although it often does. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 16, 2024, to make a final decision on Geron's New Drug Application (NDA) for imetelstat.
Imetelstat is a novel telomerase inhibitor aimed at addressing the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies. It has been granted Fast Track designation by the FDA for the treatment of certain conditions related to myelodysplastic syndromes and myelofibrosis.
Geron plans to launch imetelstat commercially in the U.S. upon potential FDA approval. If approved, imetelstat would provide a new treatment option for patients with LR-MDS, a group that currently has limited therapeutic alternatives and significant unmet medical needs, particularly among difficult-to-treat subgroups.
This article is based on a press release statement from Geron Corporation.
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