FDA Approves Soliris (Eculizumab) for Neuromyelitis Optica Spectrum Disorder

June 27, 2019

Complement inhibitor is the first treatment for the rare autoimmune disorder.

The FDA has approved Soliris (eculizumab, Alexion) as the first-ever treatment for neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. 

Originally approved in 2007, eculizumab is a complement inhibitor also indicated for the treatment of atypical hemolytic uremic syndrome and generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AchR) antibody positive. 

“Soliris provides the first FDA-approved treatment for neuromyelitis optica spectrum disorder, a debilitating disease that profoundly impacts patients’ lives,” says Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research in a statement. “This approval changes the landscape of therapy for patients with NMOSD. Having an approved therapy for this condition is the culmination of extensive work we have engaged in with drug companies to expedite the development and approval of safe and effective treatments for patients with NMOSD, and we remain committed to these efforts for other rare diseases.”

In a clinical trial comprising 143 patients with NMOSD who had antibodies against AQP4, treatment with eculizumab resulted in a reduction of NMOSD relapses by 94% over the course of the 48-week study. Treatment also reduced the need for hospitalizations and the need for treatment of acute attacks with corticosteroids and plasma exchange. 

For the treatment of NMOSD, eculizumab follows a dosing regimen of: 

  • 900 mg weekly for the first four weeks, 

  • 1200 mg for the fifth dose, 

  • then 12 mg every two weeks thereafter for the remainder of treatment

Eculizumab is available in 300 mg/30 mL single-dose vials. 

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Eculizumab is contraindicated for patients with unresolved serious Nesseria meningitidis infections, or patients who are not currently vaccinated against Nesseria meningitidis unless the risks of delaying eculizumab outweigh the risks of developing a meningococcal infection.

Treatment should be discontinued in patients who are being treated for serious meningococcal infections. 

Patients should be immunized with meningococcal vaccines at least two weeks prior to administering initial doses, according to eculizumab’s boxed warning. The warning also states that vaccinations may reduce, but not eliminate, the risk of meningococcal infections; thus, healthcare providers should continue to monitor patients for the development of meningococcal infections throughout the duration of treatment. 

Adverse reactions reported by NMOSD patients taking eculizumab include upper respiratory infection, nasopharyngitis, diarrhea, back pain, dizziness, influenza, arthralgia, pharyngitis, and contusion.

Full Prescribing Information  Healthcare Professionals’ Site