The Food and Drug Administration has accepted and given priority review to the approval application of a drug for Alzheimer’s disease that has been the subject of controversy.
Cambridge, Massachusetts-based Biogen and its partner, Tokyo-based Eisai, said Friday that the FDA had accepted their application for aducanumab, an amyloid beta-targeting drug developed for Alzheimer’s, with a target action date of March 7, 2021. Despite the priority review being granted, Biogen said it did not use its priority review voucher in the application. The FDA also plans to hold an advisory committee meeting for the application, though the date has not been determined.
“We look forward to working with the FDA throughout the review process and thank the thousands of clinicians, patients and caregivers who participated in our participated in our clinical trials and have accompanied us on this journey,” Biogen CEO Michel Vounatsos said in a statement. “We believe that aducanumab marks the beginning of a new era of potential treatments for Alzheimer’s disease that will inspire even more discovery and innovation to bring hope to those affected by this devastating disease.”
Aducanumab was tested in two Phase III trials, EMERGE and ENGAGE. Last year, the trials were stopped when it was initially determined that they would not be successful, leading to a near-death experience for the amyloid beta hypothesis – the idea that targeting the plaques that form in the brains of Alzheimer’s patients can treat the disease, which already had hitherto long failed to bear fruit. But a subsequent analysis of EMERGE indicated that it had met its primary endpoint of showing a statistically significant reduction in clinical decline, as measured by the Clinical Dementia Rating-Sum of Boxes score. A subset of data from ENGAGE, which still missed its primary endpoint, was determined to support the EMERGE outcome.
Analysts greeted the announcement with mixed opinions.
Cowen analyst Phil Nadeau wrote in a note to investors that he was “encouraged” by the news, as it showed the FDA’s willingness to act expeditiously on Biogen’s application – which it accepted about 30 days after the July 8 submission despite having 60 days to decide on whether or not to accept it – indicating that the agency is comfortable with the data and seriously considering approval. However, he cautioned that the agency’s opinion cannot be known for certain, adding that a skeptic could validly argue that the fast acceptance and priority review reflect unmet medical need in Alzheimer’s more than strength of data.
“We expect the speed of acceptance without a voucher and FDA comments about a speedy review to be viewed positively,” wrote another analyst, Morgan Stanley’s Matthew Harrison.
However, Baird analyst Brian Skorney, a longtime skeptic of the drug, took a dim view, writing that he does not expect approval. While the FDA’s promise of expeditious review reads positively, he wrote, the drug must still go through a review division and AdCom meeting, and he expects it is more likely the agency will require at least one more clinical trial.
“Given the history of the FDA Division of Neurology’s ability to provide sound critique of unsupportable applications, we believe they [sic] review will be negative but acknowledge that political pressure continues to play a clear role in everything at FDA these days,” Skorney wrote.
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