AstraZeneca Wins FDA Accelerated Approval for Ultomiris in Rare Kidney Disease

The US drug regulator has granted accelerated approval to Ultomiris, a rare-disease treatment from AstraZeneca (LON: AZN), for adults with IgA nephropathy who are at risk of disease progression. The FDA acted after Priority Review, its faster-than-standard track, and the Cambridge-based drugmaker confirmed it today in a stock exchange announcement.

The shares closed yesterday at 12,614p, down 0.03% from 12,618p the session before, so the approval is not yet in the price. London opens at 8am. Over three months the closing range has run from 11,460p to 13,134p.

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IgA nephropathy is a kidney disease in which inflammation damages the organ, and Ultomiris is cleared to reduce proteinuria, excess protein leaking into the urine. AstraZeneca says it is the first and only C5 complement inhibitor, a drug blocking a key immune-system protein, approved for the condition.

The approval rests on an interim analysis of the Phase III I CAN trial. Ultomiris cut proteinuria by 46.6% at week 34 against 5.6% with placebo, a placebo-adjusted reduction of 43.4%, and the company reported no new safety concerns.

The approval is conditional, and the company’s announcement says so plainly:

This indication is approved under accelerated approval based on reduction of proteinuria. It has not been established whether Ultomiris slows kidney function decline in patients with IgAN.

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Continued approval may depend on the trial’s primary endpoint, change in eGFR, a measure of kidney filtration, at week 106. The trial enrolled about 510 participants.

Marc Dunoyer, chief executive of Alexion, AstraZeneca’s rare-disease unit, said the approval is Ultomiris’s fifth indication. The disease affects more than 200,000 people in the US, according to company data, and Japan and other countries are reviewing filings.

Fuller I CAN results are due at the ASN Kidney Week conference in Denver tomorrow. AstraZeneca’s third-quarter results are scheduled for next Friday, the 30th of October.