AstraZeneca gets Japenese nod for Ultomiris to treat generalised myasthenia gravis

New Delhi: AstraZeneca has announced that Ultomiris (ravulizumab) has been approved in Japan for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy (IVIg) or plasmaphaeresis. Japan's Pharmaceuticals and Medical Devices Agency also indicated that Ultomiris can be considered for patients who cannot receive high-dose IVIg or plasmaphaeresis due to complications, adverse reactions or other limiting factors.

The approval of the first and only long-acting C5 complement inhibitor by the Japanese Ministry of Health, Labour and Welfare (MHLW) was based on positive results from the CHAMPION-MG Phase III trial, which showed Ultomiris was superior to placebo in the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26, a patient-reported scale that assesses patients' abilities to perform daily activities. Additionally, in prolonged follow-up results from the open-label extension, clinical benefit of Ultomiris was observed through 60 weeks.

gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. The diagnosed prevalence of gMG in Japan is estimated at approximately 22,000.

Hiroyuki Murai, MD, PhD, Professor and Chairman, Department of Neurology, School of Medicine, International University of Health and Welfare, Narita, Japan, said: "C5 inhibition is a proven approach to manage gMG. The approval of Ultomiris is an important advance for the gMG community in Japan, offering patients and physicians a new long-acting C5 inhibitor that has demonstrated sustained improvement in activities of daily living through 60 weeks, with fewer infusions per year over current treatment."

Marc Dunoyer, Chief Executive Officer, Alexion, said: "We are pleased that Ultomiris is now approved in Japan for adults with gMG, a disease that may impact their ability to work, meet family obligations and live their lives fully. The approval speaks to the strength and consistency of Ultomiris clinical data as demonstrated in the global CHAMPION-MG Phase III trial. We look forward to bringing this treatment option to people living with gMG in Japan as part of our broader strategy to expand global access to our medicines."

In CHAMPION-MG, the safety profile of Ultomiris was comparable to placebo and consistent with that observed in Phase III trials of Ultomiris in paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS).

Results from the CHAMPION-MG trial were published online in NEJM Evidence and presented at the 2022 American Academy of Neurology Annual Meeting in April.

Ultomiris was approved in the US for adults with anti-AChR antibody-positive gMG in April, and regulatory reviews are ongoing in additional countries. It was recently recommended for marketing authorisation in the European Union as an add-on to standard therapy for the treatment of adult patients with gMG who are anti-AChR antibody-positive.

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