AstraZeneca says FDA issued a complete response letter for Ultomiris sBLA

The U.S. Food and Drug Administration has issued a complete response letter, or CRL, regarding the supplemental Biologics License Application, or sBLA, for long-acting C5 complement inhibitor Ultomiris for the treatment of adult patients with neuromyelitis optica spectrum disorder, or NMOSD, who are anti-aquaporin-4 antibody positive, AstraZeneca announced. “The CRL did not request additional analysis or reanalysis of the Phase III CHAMPION-NMOSD trial data included in the sBLA submission and did not raise concerns about the efficacy or safety data from the trial. The FDA requested modifications to enhance the Ultomiris Risk Evaluation and Mitigation Strategy to further validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics prior to treatment. 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. Ultomiris is currently approved for the treatment of certain adults with NMOSD in the European Union, Japan and other countries. Ultomiris is approved by the FDA for the treatment of adult patients with generalised myasthenia gravis who are anti-acetylcholine receptor Ab+, and certain adults and children with paroxysmal nocturnal haemoglobinuria or atypical haemolytic uraemic syndrome,” the company stated.