FDA requests modifications to safety program for AZ’s Ultomiris

The FDA has issued a complete response letter to AstraZeneca for its supplemental biologics license application of Ultomiris to treat adults with neuromyelitis optica spectrum disorder who are anti-aquaporin-4 antibody positive.

According to a release from AstraZeneca, the letter from the regulatory body is related to its request for modifications to enhance the Ultomiris (ravulizumab-cwvz) Risk Evaluation and Mitigation Strategy (REMS) to further validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics prior to treatment.

The letter did not request additional analysis or reanalysis of the data from the phase 3 CHAMPION-NMOSD trial included with the license application, nor did it raise concerns about safety or efficacy data.

Ravulizumab had previously been approved by the FDA for adults with generalized myasthenia gravis who are anti-acetylcholine receptor (AChR) Ab+, as well as for certain adults and children with paroxysmal nocturnal hemoglobinuria or atypical hemolytic uremic syndrome. Ravulizumab is also approved for NMOSD in the European Union, Japan and other countries.

Alexion, AstraZeneca Rare Disease plans to work with the FDA to address the REMS modifications to release Ravulizumab to U.S. adults living with NMOSD as quickly as possible, according to the release.