AstraZeneca’s (AZ) Ultomiris (ravulizumab-cwvz) has been approved by the US Food and Drug Administration (FDA) to treat neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease estimated to affect approximately 6,000 adults in the US.
The approval means the C5 complement inhibitor is now authorised for use in adults with anti-aquaporin-4 antibody-positive NMOSD, which accounts for about three-quarters of all cases of the disease.
NMOSD affects the central nervous system, including the spine and optic nerves, and the majority of patients experience unpredictable relapses characterised by a new onset of neurologic symptoms or worsening of existing neurologic symptoms.
The FDA’s decision on Ultomiris, which is administered intravenously every eight weeks following a loading dose, was supported by positive results from the late-stage CHAMPION-NMOSD trial comparing the therapy to an external placebo arm.
The study met its primary endpoint of time to first on-trial relapse, with zero relapses observed among Ultomiris patients over a median treatment duration of 73 weeks.
Both the safety and tolerability of Ultomiris in the CHAMPION-NMOSD trial were consistent with previous clinical studies and real-world use, and no new safety signals were observed.
Sean Pittock, lead primary investigator in the study, said: “C5 inhibition has been proven to offer efficacy in reducing the risk of NMOSD relapses by blocking the complement system, a part of the immune system, from attacking healthy cells in the spinal cord, optic nerve and brain.
“With [the FDA’s] approval, patients now have the option of a long-acting C5 inhibitor treatment that showed zero relapses in the pivotal CHAMPION-NMOSD trial, supporting the primary goal of relapse prevention in treating NMOSD.”
Ultomiris is already approved for certain adults with NMOSD in the EU and Japan, and AZ has outlined that regulatory reviews are “ongoing” in additional countries. The therapy also holds authorisations for use in generalised myasthenia gravis, paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome.
Commenting on the latest approval, Marc Dunoyer, chief executive officer of Alexion, AZ’s rare disease unit, said: “Alexion has been at the forefront of innovation in NMOSD, striving to offer patients a future without fear of life-altering or even fatal relapses.
“Building on the established efficacy of C5 inhibition for people living with anti-aquaporin-4 antibody-positive NMOSD, we are proud to deliver a transformative, long-acting treatment option that has the potential to eliminate relapses with a convenient dosing schedule every eight weeks.”
