REMS Modification Delays Approval of Ravulizumab for NMOSD

The Food and Drug Administration (FDA) has issued a Complete Response Letter to AstraZeneca regarding the supplemental Biologics License Application for ravulizumab-cwvz for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 antibody positive.

Ravulizumab is a long-acting C5 complement inhibitor currently marketed under the brand name Ultomiris^® for the treatment of generalized myasthenia gravis, paroxysmal nocturnal hemoglobinuria, and atypical hemolytic uremic syndrome. The Company is seeking approval for the NMOSD indication based on data from phase 3 CHAMPION-NMOSD trial (ClinicalTrials.gov Identifier: NCT04201262).

According to AstraZeneca, the FDA’s letter is requesting modifications to the Ultomiris Risk Evaluation and Mitigation Strategy (REMS) “to further validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics prior to treatment.” The prescribing information for Ultomiris includes a Boxed Warning regarding the risk for serious meningococcal infections that have developed in patients who have received treatment.

Additional analysis of the CHAMPION-NMOSD trial will not be required as there were no concerns regarding efficacy or safety. Results from the study showed that ravulizumab met the primary endpoint demonstrating a statistically significant and clinically meaningful reduction in the risk of relapse compared with the external placebo arm.

“Alexion, AstraZeneca Rare Disease is working closely with the FDA regarding next steps for the REMS modifications and remains committed to bringing Ultomiris to people living with NMOSD in the US as quickly as possible,” the Company said in a press release.

This article originally appeared on MPR