The EC approval follows a positive opinion granted by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP), based on results from the Phase 3 CHAMPION-MG trial in 175 adults with gMG
Ultomiris approved in Europe to treat gMG in adults. (Credit: Business Wire)
AstraZeneca has received the European Commission (EC) approval for Ultomiris (ravulizumab) to treat a type of generalised myasthenia gravis (gMG) in adults.
Ultomiris is a humanised monoclonal antibody complement inhibitor that works by inhibiting the C5 protein in the terminal complement cascade of the immune system.
The drug is indicated as an add-on to standard therapy to treat gMG in adults, who are anti-acetylcholine receptor (AChR) antibody-positive.
It is being developed by Alexion, AstraZeneca Rare Disease, AstraZeneca’s business unit focused on treatments for rare diseases.
The gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease, characterised by loss of muscle function and severe weakness.
AstraZeneca said that the EC approval marks the first and only regulatory approval for a long-acting C5 complement inhibitor for the treatment of gMG in Europe.
Alexion chief executive officer Marc Dunoyer said: “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.
“Alexion’s pioneering leadership in complement science has affirmed C5 inhibition as a proven approach for managing this debilitating disease.
“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.”
The EC approval follows the granting of a positive opinion by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP).
The positive recommendation was based on results from the Phase 3 CHAMPION-MG trial, which evaluated the safety and efficacy of Ultomiris in 175 adults with gMG.
In the study, Ultomiris showed a superior change in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score compared to placebo, which is the primary endpoint.
In addition, the drug showed a safety profile that was comparable to placebo and consistent with that observed in Phase 3 trials of Ultomiris in PNH and aHUS.
CHAMPION-MG trial investigator Renato Mantegazza said: “As physicians, we see first-hand how gMG can have a debilitating impact on quality of life.
“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.”
Furthermore, Ultomiris was approved in the US and Japan, earlier this year for the treatment of gMG in certain adult populations, with regulatory reviews underway in other countries.