FDA Seeks Further Safeguards for AstraZeneca's Ultomiris Therapy, But Efficacy Not in Question

The FDA has issued a complete response letter (CRL) regarding AstraZeneca Plc's AZN supplemental Biologics License Application seeking approval for long-acting C5 complement inhibitor Ultomiris (ravulizumab-cwvz) for neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+).

Neuromyelitis optica spectrum disorder (NMOSD), previously known as Devic disease or neuromyelitis optica, occurs when the body's immune system reacts against its cells. This happens mainly in the optic nerves that connect the eye's retina with the brain and spinal cord.

The CRL did not request additional analysis or reanalysis of the Phase 3 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 (REMS) to further validate patients' meningococcal vaccination status or prophylactic administration of antibiotics before treatment.

Ultomiris is currently approved for certain adults with NMOSD in the European Union, Japan, and other countries. 

The FDA approves Ultomiris for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) Ab+, and certain adults and children with paroxysmal nocturnal hemoglobinuria or atypical hemolytic uraemic syndrome.

Price Action: AZN shares are down 0.78% at $67.11 during the premarket session on the last check Wednesday.