Advisers to the US Food and Drug Administration (FDA) on 14 March defended Geron‘s blood disorder drug, saying the benefits outweighed the risks associated with the treatment, based on data from a Phase III trial.
The FDA’s Oncologic Drugs Advisory Committee voted 12 to 2 in favour of the clinical benefit/risk profile of imetelstat based on results from the IMerge Phase III clinical trial (NCT02598661), to treat transfusion-dependent anaemia in patients with a group of blood cancers called myelodysplastic syndromes (MDS).
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The Phase III IMerge trial enrolled 178 patients across North America, Europe, the Middle East, and Asia. Imetelstat, a telomerase inhibitor, showed significantly higher red blood cell transfusion independence (RBC-TI) rates for at least eight consecutive weeks, compared to placebo, meeting its primary endpoint. Additionally, 28% of imetelstat-treated patients achieved a statistically significant improvement in the key secondary endpoint of RBC-TI at 24 weeks, compared to 3% on placebo.
The concerns leading up to the FDA’s advisory panel meeting were surrounding the high rate of cytopenias (low red blood cell counts) in patients treated with the drug, but the committee ultimately backed the drug’s efficacy and safety.
The regulatory agency will now provide a decision on the approval of the treatment by 16 June 2024 under the Prescription Drug User Fee Act.
If approved, imetelstat will be competing with Bristol Myers Squibb‘s Reblozyl (luspatercept). The drug generated $1bn last year, as per BMS’ annual report. GlobalData expects the drug to continue its upward sales trajectory and pull in $3.4bn in global sales in 2030. The FDA expanded Reblozyl’s label in August 2023 to treat the same indication. According to GlobalData’s Pharma Intelligence Center, imetelstat is forecast to generate $1.2bn in sales in 2029.
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The drug is also being investigated in a Phase III IMpactMF (NCT04576156) open-label, controlled trial to treat patients with relapsed/refractory myelofibrosis (MF). Approximately 320 subjects with intermediate-2 or high-risk MF who are relapsed after or refractory to previous JAK inhibitor treatment will be randomised to receive either imetelstat or the best available therapy (BAT).
In the announcement accompanying the backing, CEO of Geron Faye Feller said: “We are pleased with the Committee’s decision to recognise the positive clinical benefit/risk profile of imetelstat for the treatment of transfusion-dependent anaemia in adult patients with lower-risk MDS.
“There are few treatment options and significant unmet medical need remains for these patients, particularly among those with difficult-to-treat subtypes of this blood cancer.”
