阿斯利康Ultomiris获FDA加速批准用于IgA肾病，系6-K备案重复披露、无新增事实

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6-K
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a9580z.htm
ULTOMIRIS APPROVED IN US IN IGAN
a9580z
FORM 6-K
SECURITIES
AND EXCHANGE COMMISSION
Washington,
D.C. 20549
Report
of Foreign Issuer
Pursuant
to Rule 13a-16 or 15d-16 of
the
Securities Exchange Act of 1934
For the
month of October 2026
Commission
File Number: 001-11960
AstraZeneca PLC
1
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Ultomiris approved in US in IgAN
22 October 2026
Ultomiris
received accelerated approval in the
US as first and only C5 complement inhibitor to reduce proteinuria
in adults with primary immunoglobulin A
nephropathy
Based on I CAN Phase III trial results from prespecified interim
analysis in which Ultomiris demonstrated 43.4% reduction in
proteinuria vs placebo at 34 weeks
Late-breaking results from I CAN Phase III trial will be presented
at American Society of Nephrology’s Kidney Week
2026
Alexion, AstraZeneca Rare Disease’s
Ultomiris
(ravulizumab) has been approved in the US to
reduce proteinuria in adults with primary immunoglobulin A
nephropathy (IgAN) at risk for disease
progression.
This indication is approved under accelerated approval based on
reduction of proteinuria. It has not been established
whether
Ultomiris
slows kidney function decline in patients with
IgAN. Continued approval for this indication may be contingent upon
verification and description of clinical benefit in the ongoing I
CAN Phase III trial evaluating
Ultomiris
in adults with IgAN who are at risk of disease
progression.
1
The trial is ongoing and will assess
the primary endpoint of change from baseline in estimated
glomerular filtration rate (eGFR) at week 106, with data intended
to support traditional FDA approval.
2
The approval follows
Priority Review
by the US Food and Drug Administration
(FDA) based on positive results from a prespecified interim
analysis of the I CAN trial. Results showed
Ultomiris
demonstrated a 43.4% reduction in proteinuria vs
placebo at 34 weeks.
3
Ultomiris
is administered intravenously every
eight weeks in adults (based on body weight), following a loading
dose.
1
IgAN is a rare, inflammatory disease of the kidneys that can lead
to chronic kidney disease and progress to end-stage kidney disease
(ESKD). It begins when the body develops abnormal IgA proteins
resulting in immune complexes that are deposited in the kidneys
causing damage. The deposition of these complexes activates the
complement system, leading to terminal complement-driven
inflammation. This results in damage and loss of essential parts of
the kidney, including cells in the glomeruli, the part of the
kidneys that filters and cleans the blood.
4
IgAN
affects more than 200,000 people in the US.
5,6
Approximately half of people with IgAN
who have elevated protein levels in urine or reduced kidney
function are at high risk of progression to ESKD, or kidney
failure, within 10 years of diagnosis.
7-9
Richard Lafayette, MD, Professor of Medicine (Nephrology) at the
Stanford University Medical Center, Founder and Director of the
Stanford Glomerular Disease Center and external scientific
committee co-chair of the I CAN trial, said: “For many
people living with IgAN, persistent proteinuria remains a
significant challenge despite available treatments, signalling
ongoing kidney injury and heightened risk of disease progression.
With this approval of
Ultomiris
, physicians now have a C5 inhibitor that delivers
sustained terminal complement inhibition, targeting a central
driver of kidney inflammation to meaningfully reduce proteinuria in
patients living with this progressive
disease.”
Bonnie Schneider, Director & Co-Founder, IgA Nephropathy
Foundation, said: “This approval reflects the importance of
continued innovation to help improve outcomes for people living
with IgAN. With its approved use across several other rare diseases
and established safety profile,
Ultomiris
provides a new treatment option in
IgAN that gives patients – and their physicians – more
choice as they navigate their care.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “As the
first and only C5 inhibitor approved for the reduction of
proteinuria in adults with IgAN,
Ultomiris
offers a new treatment option for people living
with this serious disease. Many patients continue to face the risk
of progression to kidney failure, underscoring the need for more
targeted therapies that can slow the course of disease. This
approval marks the fifth indication for
Ultomiris
, building on Alexion’s pioneering
leadership across multiple complement-mediated rare
diseases."
In the prespecified interim analysis from the I CAN Phase III
trial,
Ultomiris
demonstrated a 46.6% reduction in 24-hour
urine protein creatinine ratio (UPCR) from baseline (95% confidence
interval [CI]: 39.0%, 53.2%) at week 34, compared to 5.6% (95% CI:
-4.9%, 15.0%) in patients receiving placebo, resulting in a
placebo-adjusted treatment effect of 43.4% (95% CI: 33.5%, 51.8%;
p<0.0001). The reduction in proteinuria was rapid, observed as
early as week 10 with
Ultomiris
(36.7% [95% CI: 30.2%, 42.6%]) and sustained
through 34 weeks, compared to placebo (8.5% [95% CI: 0.5%, 15.8%]).
These results were consistent across patient subgroups, reflecting
diverse demographic and baseline clinical characteristics and
disease severity.
3
The safety profile observed in the trial was consistent with the
known profile of
Ultomiris
and was generally well tolerated, with no
new safety concerns identified. Based on results from the
prespecified interim analysis, the most common adverse events (AE)
reported were upper respiratory tract infection (11.3%),
nasopharyngitis (9.2%) and infusion-related reactions (8.4%) in
those treated with
Ultomiris
; and upper respiratory tract infection (10.2%),
nasopharyngitis (9.0%) and hypertension (6.6%) in those receiving
placebo.
3
The interim results from the I CAN trial were
presented
at
the 2026 European Renal Association (ERA) Congress, and
late-breaking results will be presented at the American Society of
Nephrology’s Kidney Week on 23 October
2026.
3,10
Regulatory submissions based on the interim results from the I CAN
trial are also under review in Japan and additional
countries.
Notes
Immunoglobulin A Nephropathy (IgAN)
Immunoglobulin A nephropathy (IgAN) is a rare, inflammatory disease
of the kidneys that can lead to chronic kidney disease (CKD) and
progress to end-stage kidney disease (ESKD). It begins when the
body develops abnormal IgA proteins resulting in immune complexes
that are deposited in the kidneys causing damage. The deposition of
these complexes activates the complement system, leading to
terminal complement-driven inflammation. This results in damage and
loss of essential parts of the kidney, including cells in the
glomeruli, the part of the kidneys that filters and cleans the
blood. Over time, this damage impacts the ability of the kidneys to
function properly, resulting in chronic kidney disease that can
progress to end-stage kidney disease.
4
The signs and symptoms of IgAN can include blood in the urine
(haematuria), foamy urine (proteinuria), swelling in hands and feet
(oedema) and high blood pressure (hypertension).
11
Most people with IgAN do not
experience symptoms in the early stages of the disease, and
therefore, it often goes undetected until it has progressed. At
diagnosis, irreversible kidney damage may have already
occurred.
12,13
Approximately half of people with IgAN
who have elevated protein levels in urine or reduced kidney
function are at high risk of progression to ESKD, or kidney
failure, within 10 years of diagnosis.
7-9
I CAN (ALXN1210-IgAN-320)
I CAN (ALXN1210-IgAN-320) is a global, Phase III, randomised,
double-blind, placebo-controlled trial evaluating the efficacy and
safety of
Ultomiris
in adults with immunoglobulin A nephropathy
(IgAN) who are at risk of disease progression. Participants were on
stable concomitant IgAN treatment(s) consistent with standard of
care for at least three months prior to
screening.
2
Participants were randomised 1:1 to receive
either
Ultomiris
or placebo, administered intravenously for a
total of 106 weeks. Patients in the treatment arm received a
loading dose of
Ultomiris
on
Day 1, followed by regular weight-based maintenance dosing
of
Ultomiris
beginning on Day 15 and then every eight
weeks through the 106-week blinded treatment period. Patients who
completed the randomised control period had the option to enter an
open-label access period.
2
The primary endpoints are change from baseline in proteinuria based
on 24-hour urine protein creatinine ratio (UPCR) at week 34 and
change from baseline in estimated glomerular filtration rate (eGFR)
at week 106, assessed at the interim analysis and final analysis,
respectively. Key secondary endpoints for the final analysis
include reduction in 24-hour UPCR ≥ 50% from baseline at week
34, change from baseline in proteinuria based on 24-hour UPCR at
week 10, time to sustained ≥ 30% eGFR decline up to week 106
and time to first occurrence of composite kidney event up to week
106. The trial was designed to enrol approximately 510 participants
from 28 countries across North America, South America, Europe, Asia
and Australia.
2
Ultomiris
Ultomiris
(ravulizumab),
the longest-acting C5 complement inhibitor, provides immediate,
complete and sustained complement inhibition. The medication works
by inhibiting the C5 protein in the terminal complement cascade, a
part of the body’s immune system. When activated in an
uncontrolled manner, the complement cascade over-responds, leading
the body to attack its own healthy cells. Following a loading
dose,
Ultomiris
is
administered intravenously every eight weeks in adults, or every
four or eight weeks in paediatric patients (based on body
weight).
Ultomiris
is approved in
the US, EU, Japan, China and other countries for the treatment of
certain adults with paroxysmal nocturnal haemoglobinuria (PNH) and
is also approved for certain children with PNH in the US, EU, China
and other countries.
Ultomiris
is also approved
in the US, EU, Japan and other countries for the treatment of
certain adults and children with atypical haemolytic uraemic
syndrome (aHUS).
Additionally,
Ultomiris
is
approved in the US, EU, Japan, China and other countries for the
treatment of certain adults with generalised myasthenia gravis
(gMG).
Further,
Ultomiris
is
approved in the US, EU, Japan, China and other countries for the
treatment of certain adults with neuromyelitis optica spectrum
disorder (NMOSD).
Ultomiris
is approved in
the US and other countries for adults with primary immunoglobulin A
nephropathy (IgAN).
Ultomiris
is being
assessed as a treatment for additional indications as part of a
broad development programme.
Alexion
Alexion, AstraZeneca Rare Disease, is focused on serving patients
and families affected by rare diseases and devastating conditions
through the discovery, development and delivery of life-changing
medicines. A pioneering leader in rare disease for more than three
decades, Alexion was the first to translate the complex biology of
the complement system into transformative medicines, and today it
continues to build a diversified pipeline across disease areas with
significant unmet need, using an array of innovative modalities. As
part of AstraZeneca, Alexion is continually expanding its global
geographic footprint to serve more rare disease patients around the
world. It is headquartered in Boston, US.
AstraZeneca
AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led
biopharmaceutical company that focuses on the discovery,
development, and commercialisation of prescription medicines in
Oncology, Rare Disease, and BioPharmaceuticals, including
Cardiovascular, Renal & Metabolism, and Respiratory &
Immunology. Based in Cambridge, UK, AstraZeneca’s innovative
medicines are sold in more than 125 countries and used by millions
of patients worldwide. Please visit
astrazeneca.com
and
follow the Company on Social Media
@AstraZeneca
.
Contacts
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.
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.
References
1.
Ultomiris
®
(ravulizumab-cwvz)
US prescribing information; October 2026.
2.
ClinicalTrials.gov. A Phase 3, Randomized,
Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and
Safety of Ravulizumab in Adult Participants With Immunoglobulin A
Nephropathy (IgAN). NCT Identifier: NCT06291376.
Available
here
.
Accessed October
2026.
3.
Barratt
J, et al. Ravulizumab in IgA nephropathy: a prespecified interim
analysis of a randomized phase 3 trial (I CAN). Presented at
European Renal Association (ERA) Congress; 2026 Jun 6; Glasgow,
Scotland.
4.
Cheung CK, et al. The pathogenesis of IgA
nephropathy and implications for
treatment.
Nat
Rev Nephrol
. 2025;21(1):9-23.
doi:10.1038/s41581-024-00885-3. Epub 2024 Sep
4.
5.
AstraZeneca Data on File - Epidemiology estimates
are composed of a triangulation of different data sources including
Data Monitor, Decision Resources Group, Kantar Health, and internal
input (updated as of July 2026). Available
here
.
Accessed October 2026.
6.
DeCongelio M, et al. The incidence and prevalence
of immunoglobulin A nephropathy in the United
States.
Clin
Nephrol
.
2025;103(1):19-25.
7.
Pitcher   D, et al.  Long-term
outcomes in IgA nephropathy.
Clin J Am Soc Nephrol.
2023;18:727–38.
8.
Sim JJ, et al. CKD progression, kidney failure,
and mortality among US patients with IgA nephropathy.
Nephrology
Dialysis Transplantation.
2025;40(11): 2104–2117.
9.
Wong K, et al. Effects of rare kidney diseases on
kidney failure: a longitudinal analysis of the UK National Registry
of Rare Kidney Diseases (RaDaR) cohort.
Lancet
. 2024;403(10433):1279-1289.
10.
Lafayette
R, et al. Effects of Ravulizumab on Proteinuria and Hematuria in
IgAN: A Prespecified Interim Analysis of the Phase 3 I CAN Trial.
Presented at the American Society of Nephrology (ASN) Kidney Week
Congress; 2026 Oct 23; Denver, CO. Abstract FR-OR107.
11.
Rajasekaran A, et al. IgA Nephropathy: An
Interesting Autoimmune Kidney Disease.
Am J Med
Sci
. 2021;
361(2):176–194.
12.
Kidney Disease: Improving Global Outcomes (KDIGO)
IgAN and IgAV Work Group, et al. KDIGO 2025 Clinical Practice
Guideline for the Management of Immunoglobulin A Nephropathy (IgAN)
and Immunoglobulin A Vasculitis (IgAV).
Kidney Int
. 2025; 108(4S): S1-S71.
13.
Stamellou E, et al. IgA
nephropathy.
Nat Rev Dis
Primers
. 2023;9(1):67.
doi:10.1038/s41572-023-00476-9.
Matthew Bowden
Company Secretary
AstraZeneca PLC
SIGNATURES
Pursuant
to the requirements of the Securities Exchange Act of 1934, the
Registrant has duly caused this report to be signed on its behalf
by the undersigned, thereunto duly authorized.
AstraZeneca
PLC
Date:
22 October 2026
By: /s/
Matthew Bowden
Name:
Matthew Bowden
Title:
Company Secretary