Ultomiris Approved in Europe for Adults With AChR-positive gMG

1st long-acting C5 complement inhibitor for gMG approved in EU

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Ultomiris approved in Europe | Myasthenia Gravis News | stamp of approval illustration

The European Commission has approved Ultomiris (ravulizumab) as an add-on to standard therapy for adults with generalized myasthenia gravis (gMG).

Eligible patients will be positive for antibodies targeting the acetylcholine receptor (AChR) — the most common type of MG-causing antibody.

Ultomiris is now the first long-acting C5 complement inhibitor to be approved for gMG in Europe, where an estimated 89,000 people are affected by the neuromuscular disorder, according to AstraZeneca.

The decision follows a positive recommendation for the therapy from the Committee for Medicinal Products for Human Use, which was released in July.  The European Commission’s final decision is usually consistent with that advisory committee’s judgment.

“This approval in Europe of the first and only long-acting C5 inhibitor is an important step towards realising our vision of improving the lives of people living with gMG and increasing access to Ultomiris worldwide,” Marc Dunoyer, CEO of Alexion, the AstraZeneca subsidiary that originally developed Ultomiris, said in a press release.

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Ultomiris works by binding to the C5 protein, blocking the immune system’s inflammatory complement cascade that drives autoimmunity in MG. Its mechanism of action is the same as that of the approved MG therapy Soliris (eculizumab), but Ultomiris is designed to last longer in the body, allowing for less frequent dosing — infusions every eight weeks instead of every other week.

Both medications were developed by Alexion, which is now a part of AstraZeneca.

The U.S. Food and Drug Administration issued its approval of Ultomiris earlier this year for the same indication as that in Europe.

The therapy also has been approved in Japan for patients with AChR antibodies who are not eligible for or whose symptoms are difficult to control with other treatments, including intravenous immunoglobulin or plasma exchange.

“Alexion’s pioneering leadership in complement science has affirmed C5 inhibition as a proven approach for managing this debilitating disease,” Dunoyer said. “We’re proud to offer a new treatment option that provides more convenience in dosing and has shown clinical benefit in a broader range of patients, including those who remain symptomatic despite their initial standard of care treatment.”

Ultomiris’ approvals were supported by findings from the international CHAMPION MG (NCT03920293) clinical trial. That Phase 3 trial enrolled 175 adults with gMG who were positive for anti-AChR antibodies and had not previously received a C5 inhibitor.

Participants were randomly assigned to receive Ultomiris or a placebo for about six months.

Results showed Ultomiris quickly relieved gMG symptoms and improved patients’ quality of life compared with the placebo, with these benefits lasting for more than a year.

Common side effects included diarrhea, upper respiratory tract infections, cold-like symptoms, and headache.

“As physicians, we see first-hand how gMG can have a debilitating impact on quality of life,” said Renato Mantegazza, MD, professor in the department of neuroimmunology and neuromuscular diseases at Fondazione IRCCS Istituto Neurologico Carlo Besta, in Milan, Italy, and a CHAMPION-MG trial investigator.

“Today’s approval is a major advancement for treating gMG in Europe, offering patients and physicians a new, long-acting treatment option which has shown reliable efficacy and sustained improvements in activities of daily living,” Mantegazza said.

According to AstraZeneca, regulatory reviews for Ultomiris also are underway in other countries.