Approvals

AstraZenecahasannouncedthatthe US Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvs) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).

This approval follows positive results from the phase 3 CHAMPION-NMOSD trial, which were published in the Annals of Neurology. In this trial, the drug met its primary endpoint of time to first on-trial relapse, which was confirmed by an independent adjudication committee. No relapses were reported among patients on Ultomiris with a median treatment duration of 73 weeks.

The trial demonstrated a consistent safety and tolerability profile, with no new safety signals being observed, however the most common adverse events were COVID-19, headache, back pain, arthralgia and urinary tract infection.

Marc Dunoyer, chief executive officer of Alexion, AstraZeneca’s rare disease division, commented: “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 AQP4 Ab+ 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. We are grateful to the NMOSD community for their ongoing collaboration and input, which enables us to advance science for rare diseases.”

Sean J Pittock MD, director of Mayo Clinic's Center for Multiple Sclerosis and Autoimmune Neurolog y and of Mayo's Neuroimmunology Laboratory and lead primary investigator in the CHAMPION-NMOSD trial, added: “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 today’s FDA 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.”