- EMPAVELI-treated adolescents achieved a 75% relative reduction in proteinuria within 6 months versus placebo
- EMPAVELI is the only approved treatment for adolescents with C3G and primary IC-MPGN, based on VALIANT study
-
asserted
EMPAVELI → treat → study
Publication follows a recent FDA label update to expand EMPAVELI’s indication to include reducing the loss of kidney function in patients 12 years and older, in addition to reducing proteinuria
CAMBRIDGE, Mass., Sept. 17, 2026 (GLOBE NEWSWIRE) —
asserted
Publication → follow → proteinuria
Biogen Inc. (Nasdaq: BIIB) today announced the publication of results from a prespecified adolescent subgroup analysis of the Phase 3 VALIANT study evaluating EMPAVELI® (pegcetacoplan) in the Clinical Journal of the American Society of Nephrology (CJASN).
asserted
Inc. → announce → Nephrology
The dedicated subgroup analysis was first presented at the 2025 Annual Meeting of the European Society of Paediatric Nephrology (ESPN).
asserted
analysis → present → Nephrology
“C3G and primary IC-MPGN are often diagnosed early in life, when preventing progressive kidney damage can have a meaningful long-term impact,” said Bradley Dixon, M.D., Section Chief of Pediatric Nephrology and Professor of Pediatrics, University of Colorado School of Medicine and Children’s Hospital Colorado.
asserted
Dixon → diagnose → Medicine
“The VALIANT results show substantial reductions in proteinuria and stabilization of kidney function in adolescents, consistent with what we observed in the broader population.
asserted
we → show → population
These findings are especially encouraging for young patients who may otherwise face a lifetime of progressive kidney disease leading to a potential kidney transplant.
uncertain
who → face → transplant
The analysis showed that a cohort of adolescents aged 12 to 17 years with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) treated with EMPAVELI achieved clinically meaningful reductions in proteinuria and stabilization of kidney function compared with placebo.
asserted
cohort → show → placebo
Adolescents represented nearly half of the VALIANT study population, providing one of the largest randomized datasets in this age group for C3G and primary IC-MPGN.
asserted
Adolescents → represent → C3
Among the adolescent cohort (n= 55) at Week 26, results included:
- EMPAVELI-treated adolescents achieved a 75% relative reduction in proteinuria versus placebo (95% CI: 59%-84%; nominal p<0.001), with reductions observed as early as Week 4 and continuing through Week 26.
asserted
adolescents → include → Week
- 71% of adolescents treated with EMPAVELI achieved at least a 50% reduction in proteinuria, compared with 4% receiving placebo (nominal p<0.001).
asserted
% → treat → placebo
- 57% of EMPAVELI-treated adolescents achieved both stable kidney function and at least a 50% reduction in proteinuria, compared with 4% receiving placebo (nominal p=0.002).
asserted
% → treat → placebo
The publication follows the recent FDA-approved label expansion for EMPAVELI, making it the first and only therapy approved to reduce both proteinuria and the loss of kidney function in adults and adolescents aged 12 years and older.
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publication → follow → years
The label update was based on 52-week efficacy and safety data from the VALIANT study demonstrating substantial reductions in proteinuria and sustained preservation of kidney function, as measured by estimated glomerular filtration rate (eGFR).
asserted
update → base → rate
“The results published in the Clinical Journal of the American Society of Nephrology further strengthen our confidence in the benefits EMPAVELI can provide for adolescents living with C3G and primary IC-MPGN,” said Daniel Jones, Ph.D., Vice President, Head of Global Medical Affairs for EMPAVELI at Biogen.
asserted
Jones → publish → Biogen
“Together with the recent expansion of the U.S. indication for EMPAVELI, these findings reinforce the potential for EMPAVELI to help protect patients’ kidney health.
asserted
EMPAVELI → reinforce → health
EMPAVELI was well tolerated in adolescents, with a safety profile consistent with the overall VALIANT population.
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EMPAVELI → tolerate → population
About the VALIANT Study
VALIANT (NCT05067127) was a randomized, placebo-controlled, double-blind, multicenter Phase 3 study evaluating the efficacy and safety of EMPAVELI in 124 patients aged 12 years and older with C3G or primary IC-MPGN.
asserted
VALIANT → control → C3
The trial included both adolescent and adult patients with native kidney disease or post-transplant disease recurrence.
asserted
trial → include → disease
Participants were randomized to receive EMPAVELI or placebo twice weekly for 26 weeks, followed by a 26-week open-label period in which all patients received EMPAVELI.
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patients → randomize → EMPAVELI
The primary endpoint was the change in urine protein-to-creatinine ratio (UPCR) at Week 26 compared with baseline.
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endpoint → compare → baseline
About C3 Glomerulopathy and Primary IC-MPGN
C3G and primary IC-MPGN are rare, chronic, complement-mediated kidney diseases that can lead to progressive kidney damage and kidney failure.
asserted
that → mediate → damage
The diseases frequently begin early in life.
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diseases → begin → life
Approximately half of patients with C3G are diagnosed before age 18, while the median age at diagnosis for primary IC-MPGN is approximately 21 years.
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age → diagnose → MPGN
Despite standard-of-care treatment, approximately 20% of children with C3G or primary IC-MPGN progress to kidney failure within 10 to 15 years of diagnosis.
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% → progress → diagnosis
About EMPAVELI® (pegcetacoplan)
EMPAVELI® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can contribute to the onset and progression of serious diseases.
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which → target → diseases
EMPAVELI is indicated in the United States for the treatment of adult and pediatric patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), to reduce proteinuria and the loss of kidney function.
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EMPAVELI → indicate → function
EMPAVELI is also approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
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EMPAVELI → approve → hemoglobinuria
U.S. Important Safety Information for EMPAVELI
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA
EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B.
asserted
EMPAVELI → box → pneumoniae
Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors.
asserted
infections → threaten → inhibitors
These infections may become rapidly life-threatening or fatal if not recognized and treated early.
uncertain
infections → become → ?
- Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection.
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risks → complete → infection
- Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination.
asserted
they → receive → vaccination
Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected.
asserted
infection → monitor → infections
Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS.
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EMPAVELI → cause → Strategy
CONTRAINDICATIONS
- Hypersensitivity to pegcetacoplan or to any of the excipients
- For initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated Bacteria
EMPAVELI, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B.
asserted
Infections → cause → strains
Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors.
asserted
infections → threaten → inhibitors
The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria.
asserted
initiation → contraindicate → bacteria
Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information.
asserted
that → note → information
If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible.
uncertain
who → indicate → vaccines
…and 34 more, not listed.
The Aporia
