In a recent development, advisers to the U.S. Food and Drug Administration (FDA) have voiced their support for Geron's blood disorder drug, imetelstat, stating that the benefits of the treatment outweigh the associated risks. This positive outcome was based on data obtained from a late-stage trial.

By a substantial 12-to-2 margin, the panel voted in favor of the benefits of imetelstat, an injectable drug that Geron is seeking approval to use in the treatment of transfusion-dependent anemia in patients diagnosed with myelodysplastic syndromes (MDS), a group of blood cancers.

The FDA's advisory panel meeting followed a review carried out by the agency's staff on Tuesday, which highlighted concerns over a lack of clarity regarding the drug's benefits to patients and raised multiple safety concerns. The discussion during the panel meeting heavily focused on the high rate of cytopenias, or low red blood cell count, observed in patients who were tested with imetelstat.

Geron, however, maintained that these adverse effects were not uncommon, a sentiment echoed by most panel members who acknowledged the limited treatment options currently available for patients with MDS. Dr. Ranjana Advani, a panelist who voted in favor of the drug, noted, "This is not a curable disease, and there are very few options. The community of doctors who take care of these patients know how to manage these side effects."

If approved, imetelstat will mark Geron's first commercial treatment in the market, competing directly with Bristol Myers Squibb's Reblozyl, which received FDA approval last year for the same indication. Analyst Joel Beatty from Baird estimates that imetelstat, if approved, could generate sales of $933 million by 2029, with a market opportunity projected to be four times larger compared to Reblozyl.

While the FDA advisory panel voted in favor of imetelstat, the regulatory authority had concerns regarding the lack of other benefits beyond the drug's ability to increase independence from transfusion at eight weeks, which was the main study goal. A spokesperson representing Geron at the panel meeting emphasized that "transfusion independence has been the regulatory gold standard for approvals in this patient setting."

It's important to note that while the FDA typically follows the advice of its advisory panel, the agency is not obligated to do so. A decision on the approval of imetelstat is expected to be made by the FDA by June 16, which will determine the drug's fate in the market.