Disclosures: Gault reports she is an employee with Alexion Pharmaceuticals. Hawker reports she is an employee with Genentech.
October 20, 2020
4 min read

Patients with rare NMOSD have treatment options

Three therapies have been approved by the FDA to reduce relapses in patients.

Disclosures: Gault reports she is an employee with Alexion Pharmaceuticals. Hawker reports she is an employee with Genentech.
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In the last year, three therapies were approved by the FDA to significantly reduce relapses of neuromyelitis optica spectrum disorder, a rare autoimmune inflammatory disorder that can leave patients blind or paralyzed.

Between 4,000 and 15,000 patients in the United States are estimated to have neuromyelitis optica spectrum disorder (NMOSD). Patients can be affected by attacks of optic neuritis and transverse myelitis, which can result in partial recovery and remissions. Nearly half of patients experience permanent visual impairments.

In June 2019, the FDA approved Soliris (eculizumab, Alexion Pharmaceuticals), the first therapy for the treatment of anti-aquaporin-4 (AQP4) auto­antibody-positive NMOSD. The FDA then approved Uplizna (inebilizumab-cdon injection, Viela Bio) in June 2020 and Enspryng (satralizumab-mwge, Genentech) in August 2020.


Enspryng is the only subcutaneous treatment for adults with anti-AQP4 antibody-positive NMOSD, and it can be administered by the patient or a caregiver at home following training from a health care provider. It is given every 4 weeks after an initial loading dose and is designed to target and inhibit interleukin-6 receptor activity by binding to the IL-6 receptor, Kathleen Hawker, MD, medical director manager of neuro-immunology at Genentech, told Ocular Surgery News.

Kathleen Hawker, MD
Kathleen Hawker

“The fact we can block this activity is very important. The other thing is, Enspryng specifically binds to the IL-6 receptor, and it does it in a new way where it is recycled. It unbinds and then rebinds to other IL-6 receptors over a period of time, so the duration of the drug is much longer. I’ve been treating NMOSD patients for 25 years, so this is truly very exciting,” she said.

The therapy was shown to significantly reduce NMOSD relapses in two phase 3 multicenter studies, SAkuraStar and SAkuraSky, with no major increases in safety signals in terms of serious infections, Hawker said.

Before the discovery of the anti-AQP4 antibody and the difference in pathogenesis, patients with NMOSD were typically misdiagnosed as having multiple sclerosis. Specialists recognized the differences in these patients, as many experienced permanent vision loss and did not recover like typical patients with MS, she said.

“Once the brain is damaged, you cannot go backward. Getting patients on this therapy early and preventing them from becoming disabled is crucial,” she said.

The first year of treatment requires 15 doses, according to a Genentech spokesperson.


Approximately three-quarters of patients with the disease have anti-AQP4 antibody-positive NMOSD. In these patients, NMOSD occurs when the complement system becomes activated inappropriately, which can damage the optic nerves, spinal cord or brain, Laura Gault, MD, PhD, therapeutic area head, neurology clinical development at Alexion, told OSN.

Eculizumab is a first-in-class complement inhibitor that binds to complement protein C5 and prevents the inappropriate activation of the complement system. The therapy has been shown to significantly reduce risk for relapse in patients with NMOSD, she said.

Laura Gault, MD, PhD
Laura Gault

In the phase 3 PREVENT study, 98% of those who received eculizumab were relapse-free at 48 weeks, Gault said.

“The phase 3 study of Soliris in NMOSD was the first ever randomized controlled study in NMOSD and was based on promising results from a single-center 14-patient open-label independent study conducted by the Mayo Clinic in which Soliris was shown to reduce relapse frequency,” she said.

The therapy has a boxed warning that meningococcal infections have occurred in patients treated with eculizumab. These have occurred infrequently but can become life-threatening if not treated early, Gault said.

Eculizumab is available under a Risk Evaluation and Mitigation Strategy, which requires patients to be vaccinated or receive prophylactic antibiotics against meningococcal infection before beginning treatment. Health care professionals must be certified in the REMS program to prescribe eculizumab, she said.

Patients receive a loading dose of 900 mg of eculizumab for 4 weeks and then 1,200 mg every 2 weeks.

“As a result of the strong data demonstrating Soliris’ substantial impact reducing relapses, there has been broad and rapid payer adoption of Soliris’ NMOSD coverage since the medicine’s approval in June 2019. In addition, we have multiple programs to help eligible patients access our medicines,” Gault said.


The approval of Uplizna, which received breakthrough therapy and orphan drug designations from the FDA before its approval, is based on results from the N-MOmentum trial, which included 230 patients. Inebilizumab demonstrated a 77% reduced risk for relapse in patients who were anti-AQP4 antibody positive compared with those given placebo.

Additionally, 89% of patients in the anti-AQP4 antibody-positive cohort remained relapse-free in the 6-month post-treatment period compared with 58% in a placebo cohort. The safety and tolerability profile was favorable, with the most common adverse reactions being urinary tract infections, nasopharyngitis, infusion reaction arthralgia and headache.

No benefit was seen in patients who were anti-AQP4 antibody negative.

“We are proud that Viela Bio’s first approved medicine has the potential to help thousands of patients with NMOSD, a progressive and debilitating neuroinflammatory disease. We are incredibly grateful to the patients, families and care partners who participated in and supported our research,” Bing Yao, PhD, CEO at Viela Bio, said in a press release.