AstraZeneca’s plans to expand the use of Ultomiris in the United States have encountered a setback, as the FDA issued a complete response letter regarding the Ultomiris application for neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease affecting the central nervous system.
It’s worth noting that the FDA’s rejection did not raise concerns about the safety or efficacy of Ultomiris, as the drug performed well in its phase 3 NMOSD trial known as CHAMPION-NMOSD. The FDA did not request additional analysis from the study.
The primary reason for the complete response letter is related to modifications needed for the proposed Ultomiris Risk Evaluation and Mitigation Strategy (REMS). Specifically, the FDA has asked AstraZeneca to establish a system for confirming patients’ meningococcal vaccination status or administering antibiotics prophylactically before treatment.
AstraZeneca is collaborating closely with the FDA to address these REMS modifications and is committed to bringing Ultomiris to individuals with NMOSD in the United States as expeditiously as possible. Ultomiris is already approved for the treatment of certain adults with NMOSD in regions such as the European Union and Japan.
In the United States, Ultomiris is also approved for the treatment of generalized myasthenia gravis in certain adults, as well as for specific adults and children with paroxysmal nocturnal hemoglobinuria or atypical hemolytic uremic syndrome.
Ultomiris demonstrated its efficacy in NMOSD in May 2022, showing a reduced risk of relapse in NMOSD patients who tested positive for anti-AQP4 antibodies when compared to the placebo group in a clinical trial involving its predecessor, Soliris. About 75% of NMOSD patients are positive for the AQP4 biomarker, representing approximately 4,500 individuals in the United States. Soliris received FDA approval for NMOSD in 2019 based on data from the phase 3 PREVENT trial, in which 98% of patients taking Soliris were relapse-free at 48 weeks, compared to 63% in the placebo group.
In the first half of 2023, Ultomiris generated $1.36 billion in revenue, compared to Soliris’ $1.65 billion.