Eli Lilly Focus on Phase III Trial for Alzheimer’s Treatment Following FDA Fast Lane Setback
Eli Lilly’s FDA approval journey for its developmental Alzheimer’s treatment donanemab was delayed after the regulator asked for a broader dataset.
Lilly had been aiming to receive accelerated approval based on a phase II trial that demonstrated a lowering in amyloid plaque — the protein responsible for damaging neurons in Alzheimer’s disease sufferers, which has been used as a surrogate biomarker for treatment efficacy.
Therapies can be approved under the FDA’s accelerated pathway based on biomarker evidence, provided the company conducts a confirmatory trial to demonstrate a treatment’s efficacy.
Eli Lilly had been anticipating an FDA nod, which would enable it to focus on a launch of the therapeutic later on in the year.
However, the FDA requested data from a minimum of 100 patients after at least a year of continued treatment with donanemab.
The original TRAILBLAZER-ALZ study treated patients until they reached a threshold of amyloid plaque clearance, at which point patients stopped treatment.
As a result of the FDA’s decision to decline accelerated approval, Eli Lilly will now have to file phase III clinical trial data later in 2023 for traditional approval.
Eli Lilly’s previous findings suggested that using donanemab to treat patients with early Alhezimer’s disease could lead to “modestly less cognitive and functional decline”, but the drug was associated with some adverse effects, including cerebral swelling or effusions.
Trials and Tribulations in Alzheimer's Treatment Development
Anne White, Executive Vice President and President of Lilly Neuroscience at Eli Lilly, was quoted by CNN as saying the company was committed to working alongside the FDA to ensure the treatment was developed along the ‘fastest possible path’.
“We look forward to our upcoming confirmatory TRAILBLAZER-ALZ 2 phase III results and subsequent FDA submission, which we’ve always seen as the most impactful next step for patients,” she said.
Of the patients who took part in the phase II trial, there were a limited number who stayed on donanemab for 12 months — the timeframe the FDA wants to see to assess the drug’s safety.
Dr. Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Foundation, explained that this was likely a sign of the Alzheimer’s treatment working as planned.
“The drug did what it was intended to do — remove amyloid plaques in the brain — and because of the trial’s innovative design, treatment was halted for patients once the biomarket PET scans confirmed the plaques were gone,” he said in a statement issued by the Alzheimer’s Drug Discovery Foundation.
Fillit added that while amyloid-clearing drugs were one part of the solution, there remained a pressing need to develop a new generation of drugs targeting all aspects of the ‘biology of ageing’.
Alzheimer’s disease is a fatal illness that triggers a progressive decline in memory and other aspects of cognition in sufferers.
Dementia resulting from Alzheimer’s disease is the most common form in the world, accounting for 60 to 80% of all cases.
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