AstraZeneca expands Ultomiris into rare autoimmune disease

March 26, 2024

The U.S. FDA approved a label expansion for AstraZeneca’s blockbuster complement inhibitor, Ultomiris, for the treatment of neuromyelitis optica spectrum disorder (NMOSD).

The approval makes Ultomiris the first and only long-acting C5 complement inhibitor for the treatment of adult patients with the rare and debilitating autoimmune disease that affects the central nervous system. Most people living with NMOSD experience unpredictable relapses, characterized by a new onset of neurologic symptoms or worsening of existing symptoms.

The expanded label nod was based on positive results from the phase 3 CHAMPION-NMOSD trial, where zero relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks.

AstraZeneca picked up the drug — designed as a next-gen version of blockbuster Soliris — in 2021 through its $39 billion mega buyout of Alexion. Ultomiris offers a more convenient dosing regimen than Soliris, widening the drug's market potential.

First approved in 2018 as a treatment for the rare blood disorder paroxysmal nocturnal hemoglobinuria, Ultomiris has notched subsequent approvals in hemolytic uremic syndrome (a rare condition that leads to progressive kidney failure), generalized myasthenia gravis (a chronic autoimmune disorder), and now, neuromyelitis optica spectrum disorder.

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