FDA slaps CRL on regulatory review of Ultomiris in NMOSD

The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the supplemental Biologics License Application (sBLA) for long-acting C5 complement inhibitor Ultomiris (ravulizumab-cwvz) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+).

First marketed to treat paroxysmal nocturnal hemoglobinuria in 2018, Ultomiris was developed by Alexion and became part of AstraZeneca’s (LSE: AZN) portfolio following the UK pharma major’s $39 billion purchase of the rare disease specialist in 2021.

The drug generated first-half 2023 sales of $1.36 billion, a year-on-yar rise of around 60%. AstraZeneca’s shares were down a modest 1% at £10.63 by midday following the announcement.