Ultomiris Is Approved in the US for Treating AChR-positive gMG

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The long-acting complement inhibitor Ultomiris (ravulizumab-cwvz) has been approved by the U.S. Food and Drug Administration (FDA) to treat most adults with generalized myasthenia gravis (gMG).

The approval specifically covers patients who are positive for antibodies against the acetylcholine receptor (AChR) — about 80% of gMG patients.

“With the approval of Ultomiris, we’re excited that MG patients now have another option to consider as part of their personalized treatment strategies that may offer more convenience and improve muscle weakness,” Samantha Masterson, CEO of the Myasthenia Gravis Foundation of America, said in a press release.

In MG, the immune system launches an antibody-driven inflammatory attack that interferes with the communication between nerves and muscles. Ultomiris is designed to block the activation of the complement cascade, a group of immune proteins that play a central role in the inflammatory attack.

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Ultomiris was developed by Alexion Pharmaceuticals, now part of AstraZeneca. Alexion also developed another complement-blocking therapy, Soliris (eculizumab), which was approved in 2017 to treat anti-AChR-positive gMG in the U.S. It is also approved for this indication in Europe and Japan.

“Since bringing forward the first complement inhibitor, we’ve continued to listen to the community and focused innovation on the needs of gMG patients. We’re proud to deliver on this commitment with today’s approval,” said Marc Dunoyer, CEO of Alexion. “Ultomiris, the only long-acting C5 inhibitor, will benefit a broader range of patients, including those with milder symptoms.”

The FDA’s approval of Ultomiris was supported by data from a Phase 3 trial called CHAMPION-MG (NCT03920293), which enrolled 175 adults with gMG who had never before been treated with a complement inhibitor. Participants were randomized to receive Ultomiris or a placebo for 26 weeks, or about half a year.

Results showed Ultomiris outperformed a placebo on numerous measures of disease activity and quality of life, and the benefits developed rapidly following the start of treatment. Recently announced data from the study’s ongoing open-label extension study where all participants were given Ultomiris showed it continued to significantly reduce symptoms past a year of treatment.

“Despite recent advances, managing gMG is complex. Earlier intervention can preserve function and quality of life,” said James Howard Jr, MD, a professor at the University of North Carolina School of Medicine and lead investigator in the CHAMPION-MG trial.

“This approval offers patients, including those with milder symptoms, a long-acting C5 inhibitor with early onset and reliable efficacy,” Howard added.

The most common side effects of Ultomiris in gMG patients are upper respiratory tract infections and diarrhea.

Because the medication blocks immune system activity, it may increase the risk of infection. Ultomiris carries a boxed warning for serious meningococcal infections, a bacterial illness that affects the brain and spinal cord. Patients should be vaccinated against meningococcal infection before starting Ultomiris.

The medication is only available through a Risk Evaluation and Mitigation Strategy program, which is required by the FDA to manage known or potential serious side effects associated with a particular medicine.