UPDATE 2-US FDA staff raises concerns on Geron's blood disorder drug
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Christy Santhosh and Pratik Jain
Updated Tue, March 12, 2024 at 9:55 AM CDT
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(Adds statement from staff reviewers paragraph 5 onwards, analyst comment in paragraph 9)
By Christy Santhosh and Pratik Jain
March 12 (Reuters) - The U.S. Food and Drug Administration's staff reviewers said on Tuesday they were unclear if Geron's blood disorder drug provided a clear benefit to patients in a late-stage trial and raised multiple safety concerns with the treatment.
Geron is seeking approval of the injectable drug, called imetelstat, for treating transfusion-dependent anemia in patients with a group of blood cancers called myelodysplastic syndromes (MDS).
The drug, if approved, will compete with Bristol Myers Squibb's Reblozyl, which has been approved by the FDA for the same disease indication.
Last year in January, Geron CEO John Scarlett told Reuters that the company anticipates a peak market potential of $1.2 billion for imetelstat in the United States and some key EU countries by 2030.
While Geron's drug met the main goal of increasing independence from transfusion at eight weeks in a late-stage study, the FDA's staff said the "clinical meaningfulness" of the data was unclear.
"The general consensus among MDS experts has been that only a 16-week or longer period of transfusion independence is clinically meaningful," the agency's staff said ahead of a meeting of FDA's independent advisers scheduled for Thursday.
The agency said that while the drug is believed to work by treating the underlying cancer, the study did not show a "disease-modifying effect" in either extending survival or helping drive disease remission.
It also highlighted a high rate of low red blood cell count in the late-stage study.
Wedbush analyst Robert Driscoll said it was expected that the briefing documents would lean negative and added that Geron will be able to provide a strong case against the points made by the FDA. (Reporting by Christy Santhosh and Pratik Jain in Bengaluru; Editing by Shailesh Kuber)
