Breakthrough Therapy designation is granted by the FDA as a way to accelerate drug development and review. In particular, the designation is granted to drugs or biologics for serious conditions, especially when the drugs or biologics show significant improvement over current therapies or the current standard of care. Near the end of June 2021, the FDA granted Breakthrough Therapy designation to donanemab, an investigational antibody therapy developed by Eli Lilly and Company (“Lilly”). As shared in the news release, donanemab is intended to treat patients with Alzheimer’s disease.
Donanemab
So what exactly is donanemab? This passive immunotherapy is a humanized IgG1 monoclonal antibody. It works by recognizing and identifying harmful beta-amyloid, which can form into toxic clumps or plaques in the brain. Next, donanemab triggers an immune response against these amyloid plaques, helping to remove them and slowing disease progression.
Overall, Breakthrough Therapy designation hinged on data from the Phase 2 TRAILBLAZER-ALZ clinical trial. During the trial, researchers evaluated the drug’s safety, efficacy, and tolerability in patients with early-stage Alzheimer’s. Altogether, 272 patients enrolled. Patients received either 700mg intravenous donanemab for 3 doses, followed by 1400mg for the next doses, or a placebo for 4 weeks, up to a total of 72 weeks. Researchers found that improved cognition and ability to perform daily tasks. Interested in learning more? See the data published in the New England Journal of Medicine (NEJM). Based on this data, Lilly will soon submit a Biologics License Application (BLA).
Moving forward, Lilly will also spearhead its ongoing Phase 3 TRAILBLAZER-ALZ 2 clinical trial.
Alzheimer’s Disease (AD)
Alzheimer’s disease (AD) is a progressive neurological disorder characterized by brain cell death and degeneration. While doctors are not sure exactly what causes AD, many believe it stems from a mixture of environmental and lifestyle choices, alongside genetics. Risk factors include age (65+), a family history of Alzheimer’s disease, having past head trauma, being female, or having poor sleeping or exercise habits. Because AD is progressive, symptoms often worsen over time. Currently, there is no cure for AD. Symptoms include:
- Worsening memory loss
- Difficulty speaking, walking, and swallowing
- Poor reasoning or judgment-making skills
- Difficulty planning and completing familiar tasks
- Disorientation
- Change in mood and behavior
- Malnutrition or dehydration (complications)
- Falls, fractures, or bedsores (complications)
- Pneumonia and/or aspiration (complications)
