AstraZeneca gets FDA approval for Ultomiris treatment

AstraZeneca has announced that its autoimmune disease treatment Ultomiris has been given the green light by US regulators.

The drug, which is already approved for certain patients in Japan and the EU, is to be used for the treatment of adult patients with anti-aquaporin-4 (AQP4), antibody-positive neuromyelitis optica spectrum disorder (NMOSD) – a disease that affects the central nervous system including the spine and optic nerves.

Most people with NMOSD, which affects around 6,000 people in the US, experience unpredictable relapses, AstraZeneca said, suffering neurologic symptoms or the worsening of existing neurologic symptoms, which tend to be severe and recurrent and may result in permanent disability.

The positive ruling by the US Food and Drug Administration followed positive results from the recent Champion-NMOSD phase III trial, which showed zero relapses over a median treatment duration of 73 weeks.

Marc Dunoyer, chief executive of AstraZeneca's rare disease unit Alexion, said: "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."