Science-led biopharmaceutical company AstraZeneca PLC (LSE: AZN) (STO: AZN) (Nasdaq: AZN) announced on Monday that Ultomiris, a long-acting C5 complement inhibitor, has gained FDA approval for treating adults with anti-AQP4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), offering relapse-free potential.

The CHAMPION-NMOSD Phase III trial demonstrates significant relapse risk reduction.

NMOSD, a rare autoimmune disease affecting the central nervous system, sees a diagnosed prevalence of around 6,000 adults in the US. Ultomiris's safety profile remains consistent, with no new adverse events observed.

Ultomiris, already approved in Japan and the EU for NMOSD, inhibits the complement cascade to prevent attacks on healthy cells.

AstraZeneca's rare disease-focused Alexion group leads the development of Ultomiris, aiming to serve patients with rare and devastating conditions globally.