On October 22, 2024 CEL-SCI Corporation (NYSE American: CVM) reported the potential positive impact on the clinical development of its immunotherapy Multikine (Leukocyte Interleukin, Injection)* resulting from a recent U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) meeting, a public forum (Press release, Cel-Sci, OCT 22, 2024, View Source [SID1234647321]).
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FDA advisory committees provide independent expert advice to the FDA on the safety and effectiveness of new and marketed drugs and help the agency make sound and informed decisions. Advisory committees make non-binding recommendations to the FDA, which generally follows the recommendations but is not legally bound to do so.
The September 27, 2024 ODAC meeting evaluated the use of checkpoint inhibitors on patients with various cancers. PD-L1 is the biomarker most often used for patient selection for checkpoint inhibitors, the most successful class of cancer drugs including Keytruda and Opdivo. The FDA sought the ODAC’s opinion on the following:
adequacy of PD-L1 expression as a predictive biomarker for patient selection in this patient population
differing risk-benefit assessments in different subpopulations defined by PD-L1 expression
adequacy of the cumulative data to restrict the approvals of immune checkpoint inhibitors based on PD-L1 expression
Following a thorough analysis of peer-reviewed published data, the panel of experts on the ODAC voted 10-2 and 11–1 against the risk-benefit profile for PD-L1 inhibitors in various cancers in two separate votes. Most ODAC members expressed concerns about the lack of benefit demonstrated for patients with low PD-L1 expression, while some members pointed to evidence that the use of the immune checkpoint inhibitors may add unnecessary toxicities for patients while also increasing financial burdens on patients.
Checkpoint inhibitors were an estimated $48 billion global market in 2023, with PD-L1 inhibitors representing 73% of the market. Current labeling for approved checkpoint inhibitors in the indications evaluated by the FDA’s ODAC include broad approvals for all patients, regardless of PD-L1 expression.
"The FDA advisory committee’s findings and public statement on the risk-benefit of checkpoint inhibitors based on PD-L1 expression is potentially a turning point in cancer treatment, pointing to a major treatment gap in low and negative PD-L1 patients. While the approved blockbuster checkpoint inhibitors, including Keytruda which is expected to be the biggest selling drug in 2024, work well in high PD-L1 expressing patients, to our knowledge, Multikine is the only immunotherapy that is targeted to and has shown survival benefit in the low and negative PD-L1 population. CEL-SCI has already identified in our prior study low PD-L1 expression as a predictive biomarker, representing about 70% of head and neck cancer patients, which will be used as a selection criterion for our upcoming FDA confirmatory Registration Study of Multikine in the treatment of treatment-naïve resectable locally advanced head and neck cancer," stated CEL-SCI CEO Geert Kersten.
"The FDA’s ODAC decision further supports our belief that Multikine holds strong potential in treating patients across a wider range of solid tumor cancer indications alone and in combination regimens," Kersten concluded.