Three FDA-Approved Drugs for Adults with Anti-Aquaporin-4 (AQP4) Antibody-Positive NMOSD

There are now three FDA-approved medications for maintenance in NMOSD. Anything else that is prescribed is done off-label.

In NMOSD, the likelihood of recurrent disease activity is greater than 90%. With the advent of the AQP4 antibody test, we recognize a wide variety of outcomes, but the condition can still have devastating long-term impacts. Ongoing treatment with medications that suppress the immune system and help prevent recurrence is generally necessary.

There are now THREE FDA-approved medications for maintenance in NMOSD. Anything else that is prescribed is done off-label.

Soliris®️ (eculizumab) was the first FDA-approved complement inhibitor indicated for the treatment of adults with anti-aquaporin-4 (AQP4) antibody-positive NMOSD. Alexion offers patient support through its OneSource program.

Uplizna™ (inebilizumab-cdon) was the second FDA-approved treatment for NMOSD in adult patients who are AQP4 antibody positive. Viela Bio is committed to helping patients access Uplizna and they have a team in place to support both patients and their care partners. Viela VIPs 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 visit

Enspryng™ (satralizumab-mwge) is the first and only subcutaneous treatment for adults living with AQP4 antibody positive NMOSD. 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 visit 

The three off-label therapies currently used in the US are mycophenolate mofetil (CellCept), rituximab (Rituxan), and azathioprine (Imuran).

For additional information on these medications, please review the following resources:

ABCs of NMOSD: Choosing a Long-Term Treatment Option

Drs. Benjamin Greenberg, Brian G. Weinshenker, and Stacey Clardy joined the second episode of ABCs of NMOSD, entitled “Choosing a Long-Term Treatment Option.” The podcast begins with an overview of the most common treatments of neuromyelitis optica spectrum disorder (NMOSD) and their side effects. The experts discuss the three new treatments that have been recently developed, and they discuss the differences between each treatment. They talk about factors to consider when choosing a treatment option, reasons why a person might switch treatments, and insurance implications. Finally, the experts discuss pediatric considerations for treatment, how to find research studies and trials, and supplementary treatments for NMOSD in addition to medication.

ABCs of NMOSD: Understanding Clinical Trial Results for NMOSD Therapies

Dr. Michael Levy and Dr. Elias Sotirchos joined GG deFiebre of SRNA for an ABCs of NMOSD podcast on “Understanding Clinical Trial Results for NMOSD Therapies.” The medical experts begin by explaining the steps of a clinical trial and the requirements of FDA approval. They then describe the differences between types of studies and the advantages of each type. Finally, Dr. Levy and Dr. Sotirchos discuss the three new NMOSD drugs and compare the results for each trial.

2020 RNDS Presentation: Long-term Treatments for Preventing Relapses

Dr. Michael Levy discussed the long-term treatment options used in NMOSD at our recent virtual RNDS.