New Drug Trial Data Published for Kidney Disease Linked to Immune System

New Drug Trial Data Published for Kidney Disease Linked to Immune System

A major medical journal has posted final two-year results from a phase 3 trial of iptacopan, a drug being tested in patients with IgA nephropathy, a condition where the immune system damages the kidneys.

The New England Journal of Medicine shared the findings on X, linking to the full paper from the APPLAUSE-IgAN trial. IgA nephropathy (sometimes called Berger’s disease) is a kidney condition where a protein called immunoglobulin A builds up in the kidneys and causes inflammation and scarring over time. Left untreated, it can lead to kidney failure.

Iptacopan works by blocking part of the body’s complement system — a branch of the immune response that, in people with this condition, mistakenly attacks kidney tissue. The APPLAUSE-IgAN trial is a phase 3 study, meaning it is one of the later and larger stages of clinical testing before a drug can be considered for wider approval.

The journal post does not summarise the specific numerical results in the tweet itself, and the full data sits behind the journal’s publication. But the release of 24-month data from a phase 3 trial is a formal step in the regulatory process — these results will be reviewed by medicines authorities when assessing whether the drug can be approved for routine use.

IgA nephropathy affects people of all ages and is one of the more common causes of kidney disease worldwide, according to NHS information. Symptoms can include blood in the urine, protein in the urine, and swelling in the hands and feet — though some people have no symptoms at all and are only diagnosed through routine tests.

Anyone concerned about kidney health should speak to their GP in the first instance. For urgent symptoms, NHS 111 is available by phone or online.

Key information

  • IgA nephropathy symptoms to discuss with a GP include: blood or protein in the urine, swollen ankles or feet, high blood pressure, or persistent fatigue
  • NHS 111 can advise on urgent kidney-related symptoms — call 111 or use the 111 online service
  • The APPLAUSE-IgAN full paper is published in the New England Journal of Medicine

Source: @NEJM

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