Key Findings
AstraZeneca has secured FDA accelerated approval for Ultomiris in the treatment of IgA nephropathy (IgAN). The drug is indicated to reduce proteinuria, which serves as a marker for kidney function and a surrogate marker for disease activity in patients at risk of progression. This approval follows the results of the phase 3 I CAN trial.
Technical Details
Ultomiris is a complement C5 inhibitor. In the I CAN trial, patients received a loading dose followed by infusions every eight weeks. At 34 weeks, this regimen resulted in a 43.4% reduction in proteinuria compared to the placebo group. Because the approval is accelerated, AstraZeneca must provide additional data from the I CAN trial to demonstrate that the reduction in proteinuria correlates with a slowdown in the decline of kidney function. The study is currently tracking the estimated glomerular filtration rate (eGFR) over a period of 106 weeks to support full FDA approval.
Background & Context
IgAN is a rare disorder characterized by the accumulation of antibodies in the kidneys, leading to inflammation, scarring, and potential chronic kidney disease (CKD). It is the most common primary glomerulonephritis globally, affecting between 25 and 50 people per million annually, with an estimated 200,000 patients in the US. Ultomiris is already approved for four other conditions: paroxysmal nocturnal haemoglobinuria, atypical haemolytic uraemic syndrome (HUS), generalised myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). In the first six months of the year, the drug generated revenues of $2.58 billion.
Significance & Outlook
The IgAN market is seeing a surge in activity, with six new therapies reaching the US market in the last decade. AstraZeneca is also pursuing regulatory submissions for Ultomiris in Japan and other countries based on interim I CAN results. Additionally, the company is testing the drug for haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), with regulatory filings currently in progress.
Source: https://pharmaphorum.com/news/az-grabs-fda-nod-ultomiris-rare-kidney-disease-igan
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