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FDA approves Ultomiris for IgA nephropathy

FDA approves Ultomiris for IgA nephropathy

New Capabilities

AstraZeneca's C5 complement inhibitor is the first of its class cleared to cut urine protein in the rare kidney disease

October 23rd, 2026: I CAN interim results published in NEJM and presented at Kidney Week

Overview

Updated Oct 23

The US Food and Drug Administration cleared AstraZeneca's Ultomiris on 21 October to reduce proteinuria, excess protein in the urine, in adults with primary immunoglobulin A nephropathy (IgAN). The approval is accelerated, so AstraZeneca must still show the drug preserves kidney function or risk losing the indication.

On 23 October researchers published the I CAN trial's interim results in the New England Journal of Medicine and presented them at the American Society of Nephrology's Kidney Week. Among 352 patients, Ultomiris cut 24-hour urine protein by 43.4% more than placebo at 34 weeks, with serious side effects in 4.6% of treated patients versus 9.0% on placebo and no meningococcal infections. A week-106 kidney-function analysis will decide full approval.

Why it matters

Ultomiris is the first C5 complement inhibitor for IgA nephropathy, giving patients a targeted option in a disease that often ends in kidney failure.

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Key Indicators

43.4%
Proteinuria reduction vs placebo at 34 weeks
Placebo-adjusted reduction from the I CAN Phase III interim analysis.
46.6%
24-hour urine protein reduction from baseline
For Ultomiris patients at week 34, versus 5.6% for placebo.
352
Patients in the interim analysis
Adults with IgAN in the prespecified 34-week analysis published in the New England Journal of Medicine.
5
US indications for Ultomiris
IgA nephropathy is the fifth rare disease indication for the drug.
200,000+
People in the US with IgAN
AstraZeneca's estimate of Americans living with the disease.

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People Involved

MD
Marc Dunoyer
Chief Executive Officer, Alexion (AstraZeneca Rare Disease)
Leading Alexion as Ultomiris wins its fifth US indication

Organizations Involved

AstraZeneca
AstraZeneca
Pharmaceutical company
Holds FDA accelerated approval for Ultomiris in IgAN

British-Swedish drugmaker whose rare-disease unit Alexion developed Ultomiris.
U.S. Food and Drug Administration
U.S. Food and Drug Administration
Federal regulator
Granted accelerated approval for Ultomiris in IgAN

The agency that approves and regulates drugs in the United States.

Timeline

April 2026 October 2026

3 events Latest: October 23rd, 2026 · 3 weeks ago

  1. I CAN interim results published in NEJM and presented at Kidney Week

    Latest Clinical trial results

    The 34-week analysis of 352 patients showed a 43.4% placebo-adjusted proteinuria reduction. Serious adverse events were 4.6% with Ultomiris versus 9.0% with placebo, and no meningococcal infections occurred.

  2. FDA grants accelerated approval for Ultomiris in IgAN

    Regulatory approval

    The FDA approves Ultomiris to reduce proteinuria in adults with primary IgAN at risk of progression, the first C5 inhibitor cleared for the indication.

  3. I CAN interim analysis shows proteinuria reduction

    Clinical trial results

    AstraZeneca reports Ultomiris cut urine protein by 43.4% versus placebo at 34 weeks in the Phase III I CAN trial.

Scenarios

1

I CAN trial confirms eGFR benefit, Ultomiris wins full approval

Likely Resolves by End of 2027

Discussed by: AstraZeneca, which says the ongoing I CAN trial is intended to support traditional approval

The I CAN trial's primary endpoint at week 106 is change in estimated glomerular filtration rate (eGFR), a direct measure of kidney function. If the final analysis shows Ultomiris slows eGFR decline, the FDA can convert the accelerated approval to traditional approval, giving the drug an unqualified claim to preserve kidney function.

2

I CAN misses eGFR endpoint, FDA weighs indication withdrawal

Possible Resolves by End of 2027

Discussed by: AstraZeneca's own label, which states continued approval depends on confirming clinical benefit

Accelerated approvals carry confirmatory obligations. If the week-106 eGFR endpoint fails to show meaningful kidney function preservation, the FDA could begin withdrawal proceedings for the IgAN indication, despite the proteinuria improvement.

3

Rival complement drugs win IgAN approval before Ultomiris's confirmatory data

Uncertain Resolves by End of 2027

Discussed by: Nephspace, which reviewed the two-year eGFR results for rival IgAN drugs

Tarpeyo, Filspari and Fabhalta have already converted from accelerated to full approval on two-year kidney-function data, and sibeprenlimab and atacicept reported two-year eGFR results in the New England Journal of Medicine in the same week as Ultomiris's approval. If Ultomiris's week-106 eGFR data lag, rivals can market an unqualified claim to slow kidney function decline.

Historical Context

2 moments from history that rhyme with this story — and how they unfolded.

2007

Soliris and the birth of C5 inhibition (2007)

Soliris (eculizumab), Alexion's first C5 complement inhibitor, won FDA approval in 2007 for paroxysmal nocturnal hemoglobinuria, a rare blood disease. It was the first drug of its class and opened complement inhibition as a treatment field.

Then

Soliris became the standard for a handful of rare complement-mediated diseases and made Alexion a rare-disease pioneer.

Now

The C5 mechanism became a platform, with Ultomiris engineered as a longer-acting successor that requires dosing every eight weeks instead of every two.

Why this matters now

Ultomiris extends the same C5 mechanism Alexion pioneered with Soliris into kidney disease, a new organ system for the class.

2021-2023

Proteinuria-based accelerated approvals in IgAN (2021-2023)

Tarpeyo (budesonide) and Filspari (sparsentan) won FDA approval for IgAN under the accelerated pathway, using urine protein reduction as a surrogate for kidney function. They were the first treatments specifically cleared for the disease.

Then

Proteinuria became the accepted surrogate endpoint for IgAN drug trials.

Now

Later IgAN drugs, including Ultomiris, carry confirmatory obligations to prove they preserve kidney function.

Why this matters now

Ultomiris is the latest drug to ride this regulatory path, with its fate tied to the same confirmatory-testing standard the earlier approvals faced.

Sources

(12)
+6
AstraZeneca (via Cision) Fierce Pharma Investing.com ETPharma AstraZeneca US Reuters AstraZeneca Life Science Daily Pharmaceutical Executive Nephspace WebMD NephCure