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 (ClinicalTrials.gov 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).
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. https://www.astrazeneca.com/media-centre/press-releases/2021/ultomiris-accepted-for-fda-priority-review-for-gmg.html.