In April 2022, the United States approved Ultomiris for the treatment of patients with generalized myasthenia gravis (gMG). This approval was followed, just four months later, by an approval in Japan for the same indication. Now, according to a press release from Astrazeneca, the therapy has also been approved in Europe as an add-on for adults with AChR antibody-positive gMG.
This approval hinges upon data from the Phase 3 CHAMPION-MG trial. Through the trial, researchers compared Ultomiris to a placebo. They found that Ultomiris improved quality-of-life, as well as increased patients’ ability to complete various tasks and daily activities. Ultimately, the benefits from the treatment were seen over a 60-week period, highlighting a sustained and durable response. While the treatment is relatively safe and well-tolerated, some side effects associated with Ultomiris include diarrhea, the common cold, upper respiratory tract infections, and headaches.
Ultomiris is a long-acting, humanized monoclonal antibody C5 complement inhibitor that is delivered via infusion. Its European approval for gMG is particularly powerful as it is the first approved C5 complement inhibitor within Europe as a whole. This will allow more patients to have access to a treatment option which could potentially improve their lives in a meaningful way. It may also confer benefits for those who have not responded to other standards-of-care.
Outside of gMG, Ultomiris is also approved in various countries for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
What is Generalized Myasthenia Gravis (gMG)?
To first understand generalized myasthenia gravis (gMG), you must first understand myasthenia gravis as a whole. This aptly named autoimmune neuromuscular disorder causes muscle weakness and fatigue. Generalized myasthenia gravis (gMG) occurs when someone with MG is refractory to conventional therapies (meaning that they do not respond well to these therapies, experience relapse, are not clinically stable, or develop severe side effects stemming from immunosuppressive therapies). gMG antibodies attack the neuromuscular junction and disrupt signals between nerves and muscles. As a result, people with gMG experience severe muscle weakness and other symptoms. Symptoms can (but do not always) include:
- Difficulty chewing and swallowing
- Double or blurred vision
- Droopy eyelids
- Problems with balance and coordination
- Slurred speech
- Severe and sometimes debilitating fatigue
- Weakness of the upper and lower extremities
- Respiratory failure
- Myasthenic crisis