AstraZeneca’s Ultomiris Gains FDA Approval for Rare Autoimmune Condition – NMOSD

- The U.S. Food and Drug Administration (FDA) has approved AstraZeneca's Ultomiris (ravulizumab-cwvz) for treating adult patients with neuromyelitis optica spectrum disorder (NMOSD).
- This marks the fourth indication for Ultomiris, which is designed to reduce dosing frequency from biweekly infusions to once every eight weeks during the maintenance phase.
- NMOSD is a rare, chronic autoimmune disorder of the central nervous system affecting approximately 200,000 people globally.
- Ultomiris demonstrated its effectiveness in the phase 3 CHAMPION-NMOSD trial, where no patients experienced an adjudicated relapse by the time of their last visit or after a median treatment duration of 73 weeks.
- Ultomiris led to a 98.6% reduction in the risk of relapse compared to external control.
- AstraZeneca overcame an initial FDA snub and modified its mandatory safety monitoring program (Risk Evaluation and Mitigation Strategy) before receiving approval for NMOSD.
- Competitors in this space include Roche's Enspryng and Amgen's Uplizna, which are also used to treat NMOSD.
- With this new indication, Ultomiris offers potential elimination of relapses with a dosing schedule every eight weeks.
- Ultomiris has already been approved for other autoimmune diseases like generalized myasthenia gravis (gMG).
- In 2023, Ultomiris generated nearly $3 billion in sales.
- AstraZeneca continues expanding Ultomiris into hematopoietic stem cell transplant-associated thrombotic microangiopathy and cardiac surgery-associated acute kidney injury.
- The company is also conducting midphase trials in lupus nephritis and immunoglobulin A nephropathy.