Ravulizumab gets priority review for generalized myasthenia gravis


The Food and Drug Administration (FDA) has accepted for priority review the additional biologic license application for ravulizumab-cwvz in adults with generalized myasthenia gravis (gMG).

The claim 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 affected adults. with generalized myasthenia gravis who were not previously treated with a complement inhibitor. Patients were randomized 1: 1 to receive either ravulizumab-cwvz or placebo by intravenous infusion as a single weight-based loading dose on day 1, followed by a regular weight-based maintenance dose starting on day 1. day 15 every 8 weeks for 26 weeks.

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


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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 side effects were headache, diarrhea and nausea.

A target date under the Prescription Drug User Fees Act (PDUFA) 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.

Reference

The regulatory submission for Ultomiris has been accepted as part of the US FDA’s priority review for adults with generalized myasthenia gravis. Press release. AstraZeneca. Accessed December 21, 2021. https://www.astrazeneca.com/media-centre/press-releases/2021/ultomiris-accepted-for-fda-priority-review-for-gmg.html.

This article originally appeared on MPR


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