NMOSD Therapeutics at a Glance

Below you can find answers to questions about the 3 FDA-approved medications for NMOSD and the 3 medications that are used off-label for NMOSD. You can find information about each individual medication on their dedicated page: Eculizumab, Inebilizumab, Satralizumab, Rituximab, Mycophenolate Mofetil, Azathioprine.

What other names do these drugs go by?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Soliris®	Uplizna™	Enspryng®	Rituxan Truxema MabThera	Cellcept Myfortic	Azasan Imuran

How do they work?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Eculizumab is a complement inhibitor; the complement system is a part of the immune system that aids your body in attacking foreign microbes and promoting inflammation.	Inebilizumab works by killing B-cells, a type of white blood cell felt to cause inflammation leading to injury in NMOSD. Mechanistically this drug is similar to Rituximab.	Satralizumab works by inhibiting a certain protein called interleukin-6, which normally functions to mediate communication between white blood cells and increase inflammatory immune responses.	Rituximab works by killing B-cells, a type of white blood cell felt to cause inflammation leading to injury in NMOSD. Mechanistically this drug is similar to Inebilizumab.	Mycophenolate Motefil works by decreasing the number of B and T cells, two type of white blood cells felt to cause inflammation and damage in NMO. Mechanistically this drug is similar to Azathioprine.	Azathioprine works by decreasing the number of B and T cells, two type of white blood cells felt to cause inflammation and damage in NMOSD. Mechanistically this drug is most similar to Mycophenolate Mofetil.

Who should not take these drugs?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Do not receive Eculizumab if you have a meningococcal infection or have not been vaccinated against meningitis infection unless your doctor decides that urgent treatment with Eculizumab is needed.	You should not receive Inebilizumab if you have: 1) had a life-threatening infusion reaction to Inebilizumab, 2) an active hepatitis B virus infection, or 3) active or untreated inactive (latent) tuberculosis.	Do not take Satralizumab if you: 1) are allergic to ENSPRYNG or any of the ingredients in ENSPRYNG, 2) have an active hepatitis B infection, 3) have active or untreated inactive (latent) tuberculosis.	Talk with your doctor about any potential contraindications to Rituximab.	Talk with your doctor about any potential contraindications to Mycophenolate Mofetil.	Talk with your doctor about any potential contraindications to Azathioprine.

How are they taken?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Intravascular (IV) infusion in an outpatient infusion center	Intravascular (IV) infusion in an outpatient infusion center	Injections under the skin at home	Intravascular (IV) infusion in an outpatient infusion center	Oral pill at home	Oral pill at home

How often are they generally taken?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
One infusion every two weeks; to start therapy, there is one infusion weekly for five weeks, followed by infusions every two weeks	One infusion every six months, with the exception of the first dose which is divided into two infusions (at week 0 and week 2)	One injection into the skin per month, with the exception of the first month with injections at Weeks 0, 2, and 4	One infusion every six months, with the exception of the first dose which is divided into two infusions (at week 0 and week 2)	Twice per day	Once daily

What is the typical dosage?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
900 mg every two weeks	300 mg every six months	120 mg once per month	1000 mg every six months	1000 mg two times per day	Dosage is based off of weight and white blood cell counts; typical dosing is around 2 to 3 mg/kg/day

How much do they reduce my risk of relapse?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
In the PREVENT clinical trial, Eculizumab reduced the risk of relapse by roughly 94%.	In the N-Momentum clinical trial, Inebilizumab reduced the risk of relapse by roughly 73% compared to individuals with NMOSD not on therapy.	In the clinical trial Sakurastar, Satralizumab reduced the risk of relapse by roughly 55% compared to individuals with NMOSD not on therapy.	As a clinical trial has not been performed yet on these medications for NMOSD, the estimated relapse risk decrease has not been calculated. Like many other neurologic autoimmune conditions, physicians used this medication before there were FDA approved therapies available as a general immunosuppressant. It is still considered a standard of care and potential option for treatment of NMOSD by most doctors.	As a clinical trial has not been performed yet on these medications for NMOSD, the estimated relapse risk decrease has not been calculated. Like many other neurologic autoimmune conditions, physicians used this medication before there were FDA approved therapies available as a general immunosuppressant. It is still considered a standard of care and potential option for treatment of NMOSD by most doctors.	As a clinical trial has not been performed yet on these medications for NMOSD, the estimated relapse risk decrease has not been calculated. Like many other neurologic autoimmune conditions, physicians used this medication before there were FDA approved therapies available as a general immunosuppressant. It is still considered a standard of care and potential option for treatment of NMOSD by most doctors.

What are the side effects?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
The most common side effects include headache, upper respiratory tract infections, high blood pressure, nausea, vomiting, and diarrhea. Infusion reactions may occur, ranging from flu-like symptoms, low blood pressure, hives to swelling, shortness of breath and, in more severe cases, shock. A serious infection called meningococcal meningitis may occur; this is from a bacterial infection that can cause inflammation around the brain.	There is a higher risk of infections, most commonly respiratory tract and urinary tract infections. Joint pain may occur. Infusion reactions may occur, ranging from flu-like symptoms, low blood pressure, hives to swelling, shortness of breath and, in more severe cases, shock. There can be reactivation of old infections, like hepatitis and tuberculosis, which your doctor will screen for before starting the medication.	Upper respiratory tract infections, headache, rash, joint pain, fatigue, and nausea are the most common side effects.	There is a higher risk of infections, most commonly respiratory tract and urinary tract infections. There can be reactivation of old infections, like hepatitis and tuberculosis, which your doctor will screen for before starting the medication. Infusion reactions may occur, ranging from flu-like symptoms, low blood pressure, hives to swelling, shortness of breath and, in more severe cases, shock.	Diarrhea and abdominal cramps are the most common. Blood cell numbers can be decreased, raising the risk of infections. There is a small risk of blood and skin cancers.	A flu like illness upon starting the medication, which improves with time. Other side effects include stomach upset, nausea, and vomiting. Blood cell numbers can be decreased, raising the risk of infections. There is a small risk of blood and skin cancers.

What should I do to prepare for taking these drugs?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Receive a meningococcal vaccine at least two weeks before starting this medication; speak with your doctors about which vaccine is best for you.	Your doctor should check your blood work for cell counts, liver enzymes, proteins called immunoglobulins, tuberculosis, and hepatitis	Your doctor should check your blood work for cell counts, liver enzymes, tuberculosis, and hepatitis	Your doctor should check your blood work for cell counts, liver enzymes, tuberculosis, and hepatitis	Your doctor should check your blood work for cell counts and liver enzymes. You should have a skin examination by a dermatologist.	Your doctor should check your blood work for cell counts and liver enzymes. You should have a skin examination by a dermatologist.

What ongoing monitoring should occur when taking these drugs?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
NMOSD patients receiving Soliris should be monitored for early signs of meningococcal infection. No ongoing monitoring is recommended following discontinuation of Soliris.	Yearly tuberculosis and hepatitis screening. Your doctor may monitor your blood counts, including B-cell count, to determine if the drug is still working in your body.	Yearly tuberculosis and hepatitis screening. Your blood count needs to be checked six weeks after starting the medication to check for the neutrophil level. Liver function tests need to be checked monthly for the first three months, then every three months after that for the first year of therapy.	Yearly tuberculosis and hepatitis screening. Your doctor may monitor your blood counts, including B-cell count, to determine if the drug is still working in your body.	You should have a skin examination by a dermatologist yearly. Your doctor should check your blood counts and liver enzymes at least twice per year.	You should have a skin examination by a dermatologist yearly. Your doctor should check your blood counts and liver enzymes at least twice per year.

Who makes these drugs?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Eculizumab is produced by Alexion.	Inebilizumab is produced by Horizon Therapeutics.	Satralizumab is produced by Genentech (a member of Roche).	Rituxan is produced by Genentech (a member of Roche). MabThera is produced by Roche. Truxema, a biosimilar of Rituximab, is produced by Teva.	Cellcept is produced by Genentech (a member of Roche). Myfortic is produced by Novartis.	Azathioprine is produced by Pharmaceutics International.

How can I get help paying for them?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Alexion offers patient support through their OneSource program	Horizon Therapeutics is committed to helping patients access Uplizna, and they have a team in place to support both patients and their care partners. Horizon By Your Side is a complimentary support program for patients, care partners and healthcare professionals. Case managers will help navigate insurance and financial assistance needs where appropriate. For more information on this program, please click here	Genentech is committed to helping patients access the medicines prescribed by their physician. For people with NMOSD, the Enspryng Access Solutions team is available to answer questions, provide product education, injection training and help families understand insurance coverage and navigate appropriate financial assistance options to start and stay on Enspryng. Patients can call 1-844-NSPRYNG (844-677-7964) to speak to a Patient Navigator or click here click here	You can work with your doctor or local hospital’s social work department to try to get assistance covering these medications	You can work with your doctor or local hospital’s social work department to try to get assistance covering these medications	You can work with your doctor or local hospital’s social work department to try to get assistance covering these medications

Can pregnant people take these drugs?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
There is no FDA pregnancy category assigned to Eculizumab. Eculizumab has been used safely for the treatment of other medical conditions during pregnancy on an individual basis, but has not been studied in large groups of pregnant people. It is unclear if this medication is generally safe for use in pregnancy and should be discussed with your doctor.	There is no FDA pregnancy category assigned to Inebilizumab. Studies have shown that Inebilizumab can cross the placenta and decrease fetal white blood cells, similar to Rituximab. It is unclear if this medication is safe for use in pregnancy and should be discussed with your doctor.	There is no FDA pregnancy category assigned to Satralizumab. There has been no harm in animal studies, however no human studies have been performed. It is unclear if this medication is safe for use in pregnancy and should be discussed with your doctor.	Rituximab is a pregnancy category C medication. This may be a medication that can be taken before conception if planned but should be discussed on an individual basis with your physician. Babies of pregnant people taking Rituximab can have low B-cell counts, which may require monitoring.	No. Mycophenolate Modafinil is pregnancy category D, meaning this medication is not safe for use during pregnancy. It is associated with increased risks of pregnancy loss and congenital malformations.	No. Azathioprine is pregnancy category D, meaning this medication is not safe for use during pregnancy. It is associated with increased risks of pregnancy loss and congenital malformations.

Clinical trial information

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
In the PREVENT clinical trial, 143 AQP-4 antibody positive individuals were studied. They received either Eculizumab or placebo, however both groups were allowed to continue other immunosuppressive medications (steroids, azathioprine, mycophenolate modafinil, etc).	In the N-Momentum clinical trial, 230 individuals were studied. They were both positive and negative for the AQP-4 antibody. Individuals taking the drug were compared to individuals not taking any drug, with slightly better response in seropositive participants with a 77% reduction in relapse risk.	In the clinical trial Sakurastar, 95 individuals were studied. They were both positive and negative for the AQP-4 antibody. Individuals taking the drug were compared to individuals not taking any drug, with seropositive individuals responding much better than seronegative individuals.	Studies investigating Rituximab have largely been retrospective and not placebo-controlled trials, but have indicated reductions in relapse rates for those with NMOSD.	Studies investigating Mycophenolate Mofetil have largely been retrospective and not placebo-controlled trials, but have indicated reductions in relapse rates for those with NMOSD.	Studies investigating Azathioprine have largely been retrospective and not placebo-controlled trials, but have indicated reductions in relapse rates for those with NMOSD.

Will my insurance cover this?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
This will depend on your insurance company and the billing code your doctor uses. For specific questions, call the customer service phone number on the back of your insurance card with the name of the drug in question, as well as ICD (diagnostic) code your doctor uses.	This will depend on your insurance company and the billing code your doctor uses. For specific questions, call the customer service phone number on the back of your insurance card with the name of the drug in question, as well as ICD (diagnostic) code your doctor uses.	This will depend on your insurance company and the billing code your doctor uses. For specific questions, call the customer service phone number on the back of your insurance card with the name of the drug in question, as well as ICD (diagnostic) code your doctor uses.	This will depend on your insurance company and the billing code your doctor uses. For specific questions, call the customer service phone number on the back of your insurance card with the name of the drug in question, as well as ICD (diagnostic) code your doctor uses.	This will depend on your insurance company and the billing code your doctor uses. For specific questions, call the customer service phone number on the back of your insurance card with the name of the drug in question, as well as ICD (diagnostic) code your doctor uses.	This will depend on your insurance company and the billing code your doctor uses. For specific questions, call the customer service phone number on the back of your insurance card with the name of the drug in question, as well as ICD (diagnostic) code your doctor uses.

Are they FDA approved for NMOSD?

Eculizumab	Inebilizumab	Satralizumab	Rituximab	Mycophenolate Mofetil	Azathioprine
Yes	Yes	Yes	No, it is used off-label	No, it is used off-label	No, it is used off-label