The company said it will file a marketing application later this year for the drug under the FDA’s accelerated approval pathway.
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Eli Lilly’s investigational antibody therapy, donanemab, has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration for Alzheimer’s disease (AD). The company said it will file a marketing application later this year for the drug under the FDA’s accelerated approval pathway.
The agency was granted Breakthrough Therapy designation for donanemab based on data from Eli Lilly’s Phase II TRAILBLAZER-ALZ study. This randomized, multi-center trial evaluated the safety and efficacy of the antibody agent in patients with early and symptomatic AD.
A total of 272 patients in the TRAILBLAZER-ALZ trial were randomly assigned to either donanemab or placebo. Researchers compared the two arms in regard to the change in the Integrated Alzheimer’s Disease Rating Scale (iADRS) from baseline to 76 weeks.
The findings from this study, which were presented at the 15thth International Conference on Alzheimer’s and Parkinson’s Diseases 2021 and simultaneously published in the New England Journal of Medicine, showed donanemab significantly slowed the decline of the iADRS.
Despite these promising results, however, mixed findings for the secondary endpoints muddied the clarity on the therapy’s full-reaching benefits. For instance, patients in the donanemab arm demonstrated numerically better performance on several dementia rating scales compared with those in the placebo group, but the differences between the two arms weren’t statistically significant in all cases.
According to a statement made by Eli Lilly, the company said it plans to submit a biologics license application (BLA) for the therapy under the accelerated approval based on the TRAILBLAZER-ALZ findings. The company also stated it is currently evaluating donanemab’s safety, tolerability and efficacy in the Phase III TRAILBLAZER-ALZ 2 trial (NCT04437511).
Eli Lilly faces competition from Biogen’s recently approved AD drug Aduhelm (aducanumab), another antibody that is also aimed at targeting amyloid. Other up-and-coming contenders include Eisai and Biogen’s lecanemab as well as Roche’s gantenerumab.
The recent approval of Biogen’s Aduhelm was based on clinical evidence showing the drug cleared plaque-forming beta-amyloid protein clumps in the brain of a patient with AD. However, the clinical evidence didn’t necessarily show whether the drug could slow progression of disease after clearing these plaques.
Several controversies have clouded the Aduhelm approval, including the whopping price tag of $56,000 a year. Given the price point and the lack of robust clinical data showing Aduhelm slows AD progression, three members of the FDA advisory panel resigned from their posts to protect the approval.
A watchdog organization recently called for more FDA officials to resign in an eight-page letter to the U.S. Department of Health and Human Services Secretary Xavier Becerra. The organization, Public Citizen, said acting Commissioner Janet Woodcock and other agency officials involved in the aducanumab approval should resign from their posts immediately.
Additionally, the organization stated Patrizia Cavazzoni, the Center for Drug Evaluation and Research Director, and Billy Dunn, the CDER’s Office of Neuroscience Director, should also resign from their positions following the approval decision. Public Citizen also noted that the per-year price tag for the therapy threatens Medicare’s financial stability.
Public Citizen said in its letter, “The primary beneficiaries of the agency’s action are Biogen and its shareholders, who undoubtedly are ecstatic about their soon-to-be-reaped windfall profits from sales of the company’s exorbitantly priced but ineffective drug.”
