AstraZeneca's new indications for major complement C5 inhibitors receive FDA priority review qualification

Article source: Medical Rubik's Cube Info

Author: Shi Bei

On December 21, AstraZeneca announced that it had acquired a supplementary license application (sBLA) for the complement C5 inhibitor Ultomiris (ravulizumab-cwvz), a leading company on the complement track, for the treatment of adult systemic myasthenia gravis (gMG).

gMG is a rare and debilitating chronic autoimmune neuromuscular disease that can cause loss of muscle function and severe weakness

Ultomiris is the first and only approved long-acting C5 complement inhibitor with immediate, complete and continuous complement inhibition

This marketing application is based on the results of a phase III clinical study of Ultomiris in the treatment of gMG patients

Ultomiris and Soliris are the two most important listed products obtained by AZ in acquiring Alexion for US$39 billion

On August 20th, AstraZeneca announced that the Phase III CHAMPION-ALS study of Ultomiris in the treatment of adult amyotrophic lateral sclerosis (ALS) was evaluated by the Independent Data Monitoring Committee (IDMC) and did not reach the clinical endpoint in the pre-specified interim analysis

In addition to ALS, Ultomiris is approved for the treatment of neuromyelitis optica spectrum disorder (NMOSD) indications with a very high probability of expansion.

Piper Sandler analyst Christopher Raymond stated in a report in February that in 2025, Alexion's global sales revenue on MG indications may reach US$3.