Myasthenia gravis is an autoimmune condition that weakens the muscles in your body. Acetylcholine is a neurotransmitter that helps your muscles move. Most people with generalized myasthenia gravis (gMG) have antibodies in their body that attack acetylcholine receptors at the neuromuscular junction, the place where nerve cells and muscle cells connect. These antibodies are called anti-acetylcholine receptor antibodies (AChR).
Ultomiris (ul-toe-meer-is) was approved in 2022 for the treatment of gMG in people who are AChR positive.
How Does It Work for gMG?
There is no cure for myasthenia gravis. It causes muscle weakness and can have these symptoms:
- Drooping of one or both eyelids
- Changes in facial expressions due to weakness in your face, jaw, or mouth
- Weakness in your arms, legs, and hands
- Difficulty swallowing
- Trouble speaking
Different people may have different symptoms, depending on how severe their myasthenia gravis is. In myasthenia gravis, the anti-AChR antibodies block the receptors on muscle cells from receiving movement signals from your nerve cells. Your body mistakes this as a problem, and your immune system (which includes your antibodies) starts attacking your muscles.
Ultomiris is an antibody that blocks a protein called C5, which contributes to muscle damage. This helps stop your immune system from attacking and killing your muscle cells.
How Will I Receive Ultomiris?
Ultomiris will be given to you by a health care provider as an IV infusion into your vein. You may first receive a starting dose, then 2 weeks later receive another dose, and then continue to receive an IV infusion every 8 weeks. Follow the instructions given to you by your health care provider on how often you should receive your dose. Your health care provider will determine how long you should receive Ultomiris. The dose of Ultomiris you get may be based on your body weight.
Call your health care provider right away if you plan on missing a scheduled dose. Try to keep all appointments to receive your dose.
How Was It Studied?
The safety and efficacy of Ultomiris was looked at in one clinical study. People with gMG who also had anti-AChR antibodies received either Ultomiris or a placebo for 26 weeks. The study looked to see the change from baseline to week 26 in the Myasthenia Gravis – Activities of Daily Living (MG-ADL) total score. The MG-ADL score looks at the impact of myasthenia gravis on daily function. The score ranges from 0 to 24, with a higher score indicating more impairment in your day-to-day activities. The average age of people in the study was 56 years old. The study included White people (73%), Asian people (18%), Black people (3%), those classified as “Other” (2%), and those classified as “Not Reported” (4%).
What Benefits Were Seen?
People who received Ultomiris had some improvement in their MG-ADL score (-3.1) compared to people using the placebo (-1.4). That means Ultomiris helped to improve some symptoms of myasthenia gravis.
How Can I Tell if It Is Working?
Ultomiris works to improve your muscle weakness due to myasthenia gravis. You may start to see some improvement in your muscle function, which can improve your physical function. In clinical studies, Ultomiris started to improve symptoms in as little as 1 week. Talk to your health care provider if you feel that your symptoms are not improving or are getting worse. Your health care provider may increase your dose, add on another medicine, or change you to a different medicine.
What Are the Possible Side Effects and How Can I Manage Them?
The most common side effects seen with Ultomiris are diarrhea, an infection in the nose or throat, a sore throat, or common cold symptoms.
Diarrhea may occur while you are receiving Ultomiris. To limit diarrhea, you can try simple steps such as eating bland foods like rice, bananas, and toast; drinking plenty of water; and lowering the amount of caffeine you are drinking. Call your health care provider if your diarrhea symptoms are not improving or are getting worse. Your health care provider may prescribe you prescription medicines to treat this or may ask you to keep over-the-counter medicines on hand.
Call your health care provider if you are having common cold symptoms. Your health care provider will determine how to best treat these symptoms and if any other prescription or over-the-counter medicines are needed.
What Type of Drug Interactions Can Happen?
Having procedures done where your plasma is taken, such as plasma exchange or plasmapheresis, or receiving IV antibodies, also known as intravenous immunoglobulin (IVIg), can lower Ultomiris blood levels and cause the medicine to not work as well.
Using medicines called neonatal Fc receptor (FcRn) blockers, such as efgartigimod, which are treatments that may be used for myasthenia gravis, can lower blood levels of Ultomiris and can cause the medicine to not work as well.
What Are Other Unique Considerations to Be Aware Of?
Ultomiris is part of a Risk Evaluation and Mitigation Strategy (REMS) program. A REMS program is required for certain medicines, such as Ultomiris, that can have serious side effects. For Ultomiris, the serious side effect is the risk of serious meningococcal infections, including meningitis, which can affect the brain and spinal cord. Your health care provider will give you a patient safety card that tells you the signs of a meningococcal infection and when you should seek emergency medical care. You may be asked to carry the patient safety card with you while you are receiving Ultomiris and for at least 8 months after you have finished treatment. You can find out more at ultomirisrems.com.
Your health care provider may ask you if you have been vaccinated with the meningococcal vaccine. If not, your provider may ask you to get vaccinated at least 2 weeks before receiving Ultomiris or prescribe you antibiotics that may help protect against meningococcal infections.
Is There a Cost Savings Program?
The drugmaker offers a savings program that may allow you to pay as little as $0 for your Ultomiris prescription. Whether you are eligible depends on if you have commercial insurance. You can find out more by visiting ultomiris.com/gmg/cost-and-savings or by calling 877-464-8586.
