AstraZeneca Ultomiris label expansion declined in U.S.
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AstraZeneca (NASDAQ:AZN) announced Wednesday that the U.S. Food and Drug Administration (FDA) declined its request to expand the label for its rare disease therapy Ultomiris to include adults with neuromyelitis optica spectrum disorder (NMOSD).
In response to the companyâs supplemental Biologics License Application (sBLA), the agency has cited the need to add changes to the drugâs safety program under the FDAâs Risk Evaluation and Mitigation Strategy (REMS).

However, in the complete response letter (CRL), the regulator has not requested additional data and has not raised concerns over the efficacy and safety data in AstraZenecaâs (AZN) Phase 3 CHAMPION-NMOSD trial used for the BLA.
The REMS modifications suggested are steps to further validate patientsâ meningococcal vaccination status or prior prophylactic use of antibiotics, the company said.
Ultomoris is already approved in the U.S. for conditions including generalized myasthenia gravis (gMG).
The C5 complement inhibitor became part of the Anglo-Swedish drugmakerâs portfolio following its $39B acquisition of U.S.-based Alexion Pharmaceuticals in 2021.



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