Newly Approved Drugs Offer New Hope in NMOSD

Randy Dotinga

March 11, 2021

While they're extraordinarily expensive, a trio of newly approved medications is providing a variety of effective treatment options for patients with neuromyelitis optica spectrum disorder (NMOSD), a neurologist told colleagues.

"Patients have a choice of different options with different types of action. It's good news," said Sean J. Pittock, MD, of the Mayo Clinic in Rochester, Minn. "If you don't stop the clinical attacks, patients can become very disabled very quickly. These medications have a significant impact in reducing the likelihood of having a clinical relapse. If you can stop the relapses, you certainly can eventually stop most – if not all – of the disability accrual."

Pittock spoke at the annual meeting held by the Americas Committee for Treatment and Research in Multiple Sclerosis and answered follow-up questions in an interview.

Treatment Advances for NMOSD

NMOSD, also known as NMO, is a relapsing autoimmune inflammatory disorder that causes recurrent optic neuritis and myelitis. It's a rare disease, affecting 0.5-10 people per 100,000, mostly women.

Several recent trials have supported the use of the drugs eculizumab (Soliris), satralizumab (Enspryng) and inebilizumab (Uplizna) in NMOSD, Pittock said, and all have received Food and Drug Administration approval to treat the condition over the past 2 years. Pittock led the PREVENT trial of eculizumab, which showed a 94% reduction of relapse risk versus placebo.

The newly approved drugs are stunningly expensive. According to Pittock, eculizumab costs $710,000 a year, while inebilizumab runs $393,000 the first year, then $262,000 a year. Satralizumab is $219,000 the first year, then $190,000 a year. Assistance programs are available, Pittock said, "and we've not had any major problems in terms of initiation."

The cost of rituximab (Rituxan), which has a history of use as an off-label treatment option, is $18,000 a year and dropping, according to Pittock. There's also new research on rituximab: In 2020, a small Japanese trial (n = 38) reported prevention of relapses compared with placebo, but Pittock cautioned that "the placebo patients seem to have a more benign course or more benign phenotypes" than the intervention group.

"There's no doubt that rituximab works, but does it work as well as the other medications that have been through more of a robust trial process?" he asked. Keep in mind, he added, that perhaps 20%-50% of patients will relapse on rituximab.

Pittock advised colleagues to consider factors like patient schedules and compliance when choosing a drug. Satralizumab is self-administered monthly, while inebilizumab and rituximab are infused every 6 months.

Progress in Anti-MOG Disease

The trials in NMOSD should spur studies of the drugs in anti–myelin oligodendrocyte glycoprotein (MOG) disease, he said. "I think we'll see a more rapid move toward phase 3 trials because of the experience with NMO. We will just have to wait and see which medications enter trial."

Anti-MOG disease, also known as MOG antibody disease (MOGAD) and anti-MOG–associated encephalomyelitis, is caused by anti-MOG antibodies. Optic neuritis is very common, and transverse myelitis can occur.

The condition "actually responds to different drugs than MS and has a different immune pathophysiology," Pittock said.

He cautioned colleagues to be aware that "the ability of the antibody to tell you whether or not the patient has the disease is less clear for MOGAD than it is for other diseases. If your patient has a low titer of MOG antibody, and their phenotype really doesn't look like [MOGAD], you really need to interpret that with significant caution."

He also highlighted a 2018 report that offers guidance about diagnosis and when MOG-IgC antibody tests are appropriate in CNS demyelinating disease.

Pittock reported numerous disclosures plus patents issued or pending.

This article originally appeared on MDedge.com, part of the Medscape Professional Network.

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