The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application for ravulizumab-cwvz in adults with generalized myasthenia gravis (gMG).

The application is supported by data from a global, multicenter, randomized, double-blind, placebo-controlled study ( Identifier: NCT03920293) that evaluated the efficacy and safety of ravulizumab-cwvz in 175 adults with generalized myasthenia gravis who were not previously treated with a complement inhibitor. Patients were randomly assigned 1:1 to receive either ravulizumab-cwvz or placebo via intravenous infusion as a single weight-based loading dose on day 1, followed by regular weight-based maintenance dosing beginning on day 15 every 8 weeks for 26 weeks.

Findings from the study showed that treatment with ravulizumab-cwvz was associated with a statistically significant change in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score and in the Quantitative Myasthenia Gravis (QMG) total score at week 26. Improvements in MG-ADL and QMG scores were observed as early as week 1 and sustained through week 52 (26 weeks randomized controlled period + 26 weeks of open-label extension).

Continue Reading

The safety profile of ravulizumab-cwvz was consistent with that observed in previous studies of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. The most frequently reported adverse events were headache, diarrhea, and nausea.

A Prescription Drug User Fee Act (PDUFA) target date has been set for the second quarter of 2022.

Ravulizumab-cwvz, a complement inhibitor, is marketed under the brand name Ultomiris and is currently approved for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy.


Ultomiris regulatory submission accepted under FDA Priority Review in the US for adults with generalised myasthenia gravis. News release. AstraZeneca. Accessed December 21, 2021.

This article originally appeared on MPR