EC Clears Eculizumab (Soliris) for Neuromyelitis Optica

Megan Brooks


August 28, 2019

The European Commission (EC) has approved an expanded indication for the humanized monoclonal antibody eculizumab (Soliris, Alexion Pharmaceuticals) to include adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody—positive and whose disease is following a relapsing course.

NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system that primarily affects women. It is characterized by relapses that are associated with increasing disability, including blindness, paralysis, and sometimes premature death. Roughly three quarters of patients with the disorder have anti-AQP4 antibodies.

The EC's approval of eculizumab for NMOSD was based in large part on results from the phase 3 multicountry PREVENT trial, which were presented at the 2019 annual meeting of the American Academy of Neurology and were simultaneously published online May 3 in the New England Journal of Medicine.

As reported at the time by Medscape Medical News, 43% of the 143 participants with AQP4 antibody—positive NMOSD who received placebo experienced adjudicated relapses, vs 3% of those who received intravenous eculizumab. For the latter group, annualized relapse rates were also lower.

"In a disease marked by unpredictable relapses that each have the potential for irreversible consequences, such as blindness or the inability to walk, the primary treatment goal is prevention of such attacks," John Orloff, MD, executive vice president and head of research and development at Alexion, said in a statement.

Nearly all patients who underwent treatment were relapse free in the phase 3 study, he added.

The safety profile of eculizumab was consistent with that seen for the drug in other clinical studies and in real-world use in its three approved indications, according to the company. The most common adverse events (AEs) observed in the PREVENT study with eculizumab were upper respiratory tract infection, headache, nasopharyngitis, and nausea.

Serious AEs, which were reported in at least one patient in either group, were pneumonia (three patients in the eculizumab group vs one patient in the placebo group) and cellulitis, sepsis, and urinary tract infection (two patients for each event in the eculizumab group vs no patient in the placebo group).

One patient who received eculizumab and concomitant supportive immunosuppressive therapy died from infectious pleural effusion. However, the patient had an extensive history of pulmonary disease and was an active smoker. No cases of meningococcal infection were observed in the study.

The US Food and Drug Administration approved eculizumab for NMOSD in June.

In the United States and Europe, eculizumab is also indicated for paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and generalized myasthenia gravis.

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