The European Commission (EC) has approved Aexion’s – part of the AstraZeneca Rare Disease group – Ultomiris (ravulizumab) in Europe as an add-on to standard therapy for generalised myasthenia gravis (gMG).
The approval is specifically for Ultomiris as a treatment for adult patients who are anti-acetylcholine receptor (AChR) antibody-positive, and the decision is a milestone in marking the first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG in Europe.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that causes a loss of muscle function and severe weakness over time. In the EU, the diagnosed prevalence of gMG is approximately 89,000.
80% of patients diagnosed with gMG are AChR antibody positive, which means they produce specific antibodies (anti-AChR). These antibodies cling to signal receptors at the neuromuscular junction (NMJ), which is the connection point between nerve cells and the muscles they control.
The binding then activates the complement system, which is key to the body’s defence against infection, leading the immune system to attack the NMJ. This causes inflammation and a breakdown in communication between the brain and the muscles.
The EC’s decision follows the positive opinion of the Committee for Medicinal Products for Human Use and is supported by results from the CHAMPION-MG phase 3 trial, which were published online in NEJM Evidence.
The results showed that 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.
Moreover, in prolonged follow-up data taken from the open-label extension, clinical benefit of Ultomiris was observed through 60 weeks.
The safety profile of Ultomiris when evaluated in the CHAMPION-MG study was comparable to placebo and consistent with that observed in phase 3 trials of Ultomiris in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome.
Marc Dunoyer, chief executive officer, Alexion, 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.”
He added: “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.”
In April 2022 Ultomiris was approved in the US and, in the same year, was then approved in Japan for certain adults with gMG.