FDA Rejects AstraZeneca's Ultomiris Expansion Plan, Citing Risk Mitigation Issues

AstraZeneca's rare disease drug Ultomiris has received a Complete Response Letter (CRL) from the FDA for its use in treating neuromyelitis optica spectrum disorder (NMOSD). The FDA has requested modifications to the drug's Risk Evaluation and Mitigation Strategy (REMS), specifically asking for enhanced checks to ensure patients have been vaccinated for meningococcal disease or will undergo preventive use of antibiotics before treatment. Ultomiris, which was first approved in the US in 2018, carries a black box warning for serious meningococcal infections.