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(Adds panel member’s comment in paragraph 7, analyst’s comment in paragraph 9)
By Pratik Jain and Christy Santhosh
March 14 (Reuters) – Advisers to the U.S. Food and Drug Administration on Thursday endorsed the benefits of Geron’s drug for blood disorders, saying it outweighed the risks associated with the treatment, based on data from a late-stage trial.
The panel, by a wide margin of 12 to 2, voted in favor of the benefits of the injectable drug, called imetelstat, stating that the associated risks and toxicities of the treatment appear to be manageable.
Geron is seeking approval to use imetelstat to treat transfusion-dependent anemia in patients with a group of blood cancers called myelodysplastic syndromes (MDS).
The FDA advisory panel meeting followed a review by agency staff on Tuesday, who noted a lack of clarity in data about benefit to patients and raised multiple safety concerns.
Much of the panel’s discussion, as well as the agency’s concerns, focused on the high rate of cytopenias, or low red blood cell counts, in patients tested with the drug.
Geron said adverse effects were not uncommon, something most panel members agreed with, given the limited current treatment options for patients.
“This is not a curable disease and there are very few options. The community of doctors who care for these patients know how to manage these side effects,” said Dr. Ranjana Advani, a panelist who voted in favor of the drug.
Imetelstat, if approved, will be Geron’s first commercial treatment on the market and will compete with Bristol Myers Squibb’s Reblozyl, which received a label expansion from the FDA last year for the same disease indication.
Baird analyst Joel Beatty projects that imetelstat, if approved, will generate $933 million in sales by 2029, and estimates the market opportunity will be four times larger compared to Reblozyl.
The US health regulator also highlighted concerns about the lack of other benefits beyond the drug meeting the study’s primary goal of increasing transfusion independence at eight weeks.
A company spokesperson present at the panel meeting noted that “transfusion independence has been the regulatory gold standard for approvals in this patient setting.”
The regulator, which normally follows the advice of its panel but is not required to do so, is expected to make a decision on the drug by June 16.
(Reporting by Pratik Jain and Christy Santhosh in Bengaluru; Editing by Krishna Chandra Eluri)