Accelerated approval application was
based on Phase 2 trial showing amyloid plaque
lowering
Complete response letter based on
limited number of patients with 12-month drug exposure data in the
accelerated approval submission; no other deficiencies were
identified
Definitive Phase 3 readout and
submission for traditional approval remain on track for mid-year
2023
INDIANAPOLIS, Jan. 19,
2023 /PRNewswire/ -- Eli Lilly and Company
(NYSE: LLY) today announced the U.S. Food and Drug Administration
(FDA) has issued a complete response letter for the accelerated
approval submission of donanemab for the treatment of
early symptomatic Alzheimer's disease due to the limited
number of patients with at least 12 months of drug
exposure data provided in the submission.
No other deficiencies in the application were
noted.
The confirmatory Phase 3 TRAILBLAZER-ALZ 2
trial remains ongoing, with topline data read-out expected in Q2
2023, and will form the basis of donanemab's application for
traditional approval shortly thereafter. Lilly will continue to
work with the FDA to evaluate the fastest pathway to make this
potential treatment option widely available to patients.
In the complete response letter to the accelerated
approval application, the FDA
specifically requested that Lilly provide data from at least 100
patients who received a minimum of 12 months of continued treatment
on donanemab. Donanemab's specificity to target deposited amyloid
plaque informed the unique clinical trial design of
TRAILBLAZER-ALZ, which allowed patients to complete their course of
treatment when they reached a predefined level of amyloid plaque
clearance. While the trial included more than 100 patients treated
with donanemab, due to the speed of plaque reduction, many patients
were able to stop dosing as early as 6 months of treatment,
resulting in fewer than 100 patients receiving 12 months of
donanemab. The FDA indicated that the data to
meet the exposure expectation would likely need to include the
unblinded controlled safety data from TRAILBLAZER-ALZ 2 upon
completion.
The safety profile of donanemab was initially reported
from the TRAILBLAZER-ALZ trial in the New England Journal of
Medicine (Mintun et al, NEJM 2021)1. The
safety profile of donanemab has remained consistent
since our accelerated approval submission.
"We look forward to our upcoming confirmatory
TRAILBLAZER-ALZ 2 Phase 3 results and subsequent FDA submission,
which we've always seen as the most impactful next steps for
patients. We anticipate this study will confirm the benefit
and safety profile we observed in the TRAILBLAZER-ALZ Phase 2 study
and believe that patients and physicians will be well served by
having the full Phase 3 data available alongside our Phase 2 data
when they need to make treatment decisions," said Anne White, executive vice president and
president of Lilly Neuroscience, Eli Lilly and Company. "We are
committed to working with the FDA to ensure the fastest possible
path to bring this potential medicine to patients in
need."
This action does not result in a change to Lilly's 2023
financial guidance.
About Alzheimer's
Disease
Alzheimer's disease is a fatal illness
that causes progressive decline in memory and other aspects of
cognition. Dementia due to Alzheimer's disease is the most common
form of dementia, accounting for 60 to 80 percent of all
cases1. There are currently over 55 million people
living with dementia around the world, with numbers expected to
increase to nearly 139 million by 20502. Over 10 million
new cases of dementia are diagnosed each year worldwide, implying
one new case every 3.2 seconds and a significant increase in the
caregiving burden placed on society and families. In the U.S.
alone, there was an increase of nearly 10 million new caregivers
from 2015 to 20203. The current annual societal and
economic cost of dementia is estimated at $1.3 trillion, an amount that is expected to
double by 2030 unless we find a way to slow the
disease2.
About Lilly
Lilly unites caring
with discovery to create medicines that make life better for people
around the world. We've been pioneering life-changing discoveries
for nearly 150 years, and today our medicines help more than 47
million people across the globe. Harnessing the power of
biotechnology, chemistry and genetic medicine, our scientists are
urgently advancing new discoveries to solve some of the world's
most significant health challenges, redefining diabetes care,
treating obesity and curtailing its most devastating long-term
effects, advancing the fight against Alzheimer's disease, providing
solutions to some of the most debilitating immune system disorders,
and transforming the most difficult-to-treat cancers into
manageable diseases. With each step toward a healthier world, we're
motivated by one thing: making life better for millions more
people. That includes delivering innovative clinical trials that
reflect the diversity of our world and working to ensure our
medicines are accessible and affordable. To learn more,
visit Lilly.com and
Lilly.com/newsroom or follow us on
Facebook, Instagram,
and LinkedIn. P-LLY
© Lilly USA,
LLC 2022. ALL RIGHTS RESERVED.
Cautionary Statement Regarding Forward-Looking
Statements
This press release contains forward-looking statements (as
that term is defined in the Private Securities Litigation Reform
Act of 1995) about donanemab as a potential treatment for people
with early symptomatic Alzheimer's disease, and reflects Lilly's
current beliefs and expectations. However, as with any such
undertaking, there are substantial risks and uncertainties in the
process of drug research, development, and commercialization. Among
other things, there is no guarantee that future study results will
be consistent with study findings to date, that donanemab will
prove to be a safe and effective treatment, or that donanemab will
receive regulatory approval. For further discussion of these and
other risks and uncertainties, see Lilly's Form 10-K and Form 10-Q
filings with the United States Securities and Exchange Commission.
Except as required by law, Lilly undertakes no duty to update
forward-looking statements to reflect events after the date of this
release.
- Mintun M, Lo A, Evans C, et al. Donanemab in Early
Alzheimer's Disease. N Engl J Med. 2021. 384:1691-1704. doi:
10.1056/NEJMoa2100708.
- Alzheimer's Association. 2022 Alzheimer's Disease
Facts and Figures.
https://www.alz.org/media/Documents/alzheimers-facts-and-figures.pdf.
Accessed May 12, 2022.
- Alzheimer's Disease International. Dementia
Statistics.
https://www.alzint.org/about/dementia-facts-figures/dementia-statistics/.
Accessed May 12, 2022.
- AARP. 2020 Report: Caregiving in the U.S.
.
https://www.aarp.org/content/dam/aarp/ppi/2020/05/full-report-caregiving-in-the-united-states.doi.10.26419-2Fppi.00103.001.pdf.
Accessed May 12, 2022.
Refer
to:
|
J.K. Wall;
jkwall@lilly.com; (317) 433-5328 (Media)
Joe Fletcher; jfletcher@lilly.com; (317) 296-2884
(Investors)
|
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SOURCE Eli Lilly and Company