Future Rare Diseases, vol.4, no.1, 2024 (Scopus)
What is this summary about?: This is a summary of a paper published in The Lancet medical journal describing the results of the NefIgArd clinical trial, which looked at Nefecon treatment in people with immunoglobulin A nephropathy (IgAN). IgAN is a kidney disease caused by a special type of antibody called galactosedeficient immunoglobulin A (gd-IgA), which collects in the kidneys. This triggers inflammation (which is the body fighting disease) and damages the kidneys over time (nephropathy), often leading to kidney failure. Nefecon is a new pill containing a medicine called budesonide and acts in the lower part of the small intestine, where the IgAN disease process is thought to start. What happened in the NeflgArd study?: NefIgArd included 364 people with IgAN who were taking the highest doses of standard of care at the time (in this case, drugs to lower blood pressure) that they were able to tolerate. They were given Nefecon or a placebo for 9 months and monitored for another 15 months. What were the results?: Compared with placebo, 9 months of Nefecon treatment slowed down the loss of kidney function, suggesting that it led to less kidney damage. These benefits were still seen after 15 months without treatment. Side effects in people taking Nefecon were manageable and were as expected for this type of drug. What do the results of the study mean?: Nefecon slows the speed at which people lose kidney function and may help protect the kidney from further damage. People with IgAN will get the most benefit from Nefecon treatment if it is started as soon as possible. Clinical Trial Registration:NCT03643965 (ClinicalTrials.gov).