Diagnosing and treating neuromyelitis optica spectrum disorder (NMO) early is essential. Because an NMO attack can lead to worsening disability, it is critical to decrease the number and severity of attacks. Until recently, preventive drugs approved by the US Food and Drug Administration (FDA) were not available to those with NMO. New drugs bring hope for better long-term treatment outcomes with NMO.1
Approved biologic drugs for NMO
Biologics are drugs made from living organisms or cells. The process of making biologics turns products made from cells into drugs that can prevent, treat, or cure diseases. Biologics are different from most traditional drugs. Traditional drugs are made from chemicals in a lab. Their ingredients are not directly derived from living cells.2
Side effects can vary depending on the specific drug you are taking. As of 2023, the FDA has approved 3 antibody-based biologic drugs for the treatment of those with AQP4 antibody (AQP4-IgG) NMO:1
Soliris® (eculizumab)
Soliris (eculizumab) is a laboratory-made antibody used to decrease the risk of an NMO attack. Soliris is a terminal complement inhibitor drug. The complement pathway is involved in protecting the body from harmful infections and is linked to inflammation. By inhibiting this pathway, Soliris decreases inflammation. Soliris is given by intravenous (through the vein, or IV) infusion, usually every 5 weeks.3,4
Enspryng™ (satralizumab)
Enspryng™ (satralizumab) is the first approved treatment in NMO that is made to block interleukin-6 (IL-6). IL-6 is a protein made by different cells in the body that plays a role in the inflammation that occurs in NMO. Enspryng blocks IL-6 from attaching to the cell surface.5,6
Enspryng is given by self-injection once per month. If your doctor prescribes this drug, you will receive injection training on the safe and correct way to take this drug.5,6
Uplizna™ (inebilizumab)
Uplizna™ (inebilizumab) works by lowering the number of the body’s B cells, which are part of your immune response. When the number of these cells is lowered, the immune system may not overreact as severely for those with NMO. Uplizna is given twice a year by IV infusion.7,8
Off-label drugs
For a drug to be approved by the FDA, a company must show data about the safety and effective use of the drug. All drugs have side effects, so “safe” does not mean “without side effects.” The FDA must decide that the drug has more benefits than risks (side effects) in order for it to gain approval.9
FDA approval takes time and requires careful evaluation. Sometimes, doctors decide an approved drug may benefit another condition that the drug was not approved to treat. This is called off-label use. It is also called off-label use if a drug is given in a different dose (such as taking 2 pills instead of 1) or in another way (like taking a liquid form instead of a pill).9
Off-label use happens when a drug has not been approved by the FDA to treat a certain condition. It means the FDA has not yet determined the drug is safe and effective when used the way you are taking it.9
Using drugs off-label is common in rare diseases. This is because there often are not many drugs available to treat these conditions. If you and your doctor decide to use a drug off-label, it is important to understand what off-label use means.
Off-label use does not mean the drug is unsafe to take. Often, it means there has not been an official clinical trial to test the drug for your specific rare disease. You should always talk to your doctor about any questions you have about your treatments.9
Some off-label and unapproved drugs commonly used to treat NMO include:10-16
- Rituxan® (rituximab), a laboratory-made antibody that targets B cells
- Mitoxantrone, an anti-cancer drug
- Campath® and Lemtrada® (alemtuzumab), which are laboratory-made antibodies
- Imuran® and Azasan® (azathioprine), which are drugs that decrease the immune response
- CellCept® (mycophenolate mofetil), a drug that decreases the immune response
- Methotrexate, a disease-modifying antirheumatic drug (DMARD)
- Actemra® (tocilizumab), a laboratory-made antibody that targets IL-6
Things to consider about preventive treatment
Preventative treatments work on your immune system to decrease inflammation. Because of this, using these immunosuppressive drugs increases your risk for infection. Some drugs have more risk than others. Talk to your doctor about your risk and the benefits of these drugs.17
You will have some blood tests and possibly some vaccines before starting immunosuppressive drugs, including biologics. The blood tests will tell your doctor if you have an infection or condition that prevents you from taking the immunosuppressive therapy.You may need vaccines to prevent you from getting certain diseases while taking a biologic or other immunosuppressive treatment.11-13,16
Immunosuppressants should not be given to anyone with an active infection or anyone allergic to those drugs.17
The complications, risks, and side effects listed here are not all that may occur with these drugs. Talk to your doctor about what to expect if you experience any changes that concern you during treatment with these preventive drugs.
Before beginning treatment for NMO, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.
