AstraZeneca has notched another indication to the belt of its rare disease therapy Ultomiris (ravulizumab) through an accelerated US approval in primary immunoglobulin A nephropathy (IgAN), a type of chronic kidney disease.
Following the US Food and Drug Administration’s (FDA) call to initially greenlight Ultomiris in IgAN, the drug will provisionally be available to patients at risk of disease progression – specifically to reduce the presence of protein in the urine.
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Ultomiris’ accelerated approval stems from the Phase III I CAN trial (NCT06291376) in which the intravenously delivered C5 complement inhibitor prompted a placebo-adjusted 43.4% drop in the presence of excess urine proteins in IgAN patients at the 34-week mark.
While the drug has already shown promise in reducing proteinuria, AstraZeneca is yet to establish whether Ultomiris can slow the functional decline of kidneys in IgAN patients. This means that Ultomiris’ full US approval in IgAN will hinge on whether the drug can demonstrate clinical benefit in the ongoing Phase III I CAN trial, in which researchers are evaluating the drug’s impact on the rate at which the kidneys filter blood.
IgAN is a rare, progressive kidney disease that leads to the damage and destruction of cells in the glomeruli, which play an essential role in the kidney’s ability to filter blood. Currently, scientists estimate that around 2.5 in every 100,000 people worldwide are living with IgAN – of which approximately half have elevated levels of protein in their urine.
With an accelerated approval secured, Ultomiris will join a competitive IgAN market, which is currently dominated by Asahi Kasei-owned Calliditas Therapeutics’ first-to-market therapy Tarpeyo (budesonide). However, Novartis’ therapy Fabhalta (iptacopan) which became the first approved complement inhibitor for this indication, is rapidly closing the gap, with the drug generating $505m in 2025 compared with Tarpeyo’s $517m in the same year.
The competition is set to heat up further, however, as there are 22 ongoing Phase III studies in IgAN, according to GlobalData’s Pharmaceutical Intelligence Center.
Recently, Roche’s potential Fabhalta rival sefaxersen met its endpoints in the Phase III IMAgINATION study (NCT05797610), while Vertex Pharmaceuticals is also seeking approval for povetacicept after the drug was successful in the late-stage RAINIER trial (NCT06564142).
As per a recent report by GlobalData, parent company of Pharmaceutical Technology, there are currently 112 molecules in the IgAN pipeline, with three in the pre-registrational phase and 40 in mid-to-late-stage development.
