Eculizumab: A Review in Neuromyelitis Optica Spectrum Disorder
Compliance with Ethical Standards
Funding The preparation of this review was not supported by any external funding.
Conflicts of interest James Frampton is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no relevant conflicts of interest
Additional
information about this Adis Drug Review can be found here
Abstract
The terminal complement protein (C5) inhibitor
eculizumab (Soliris®) is the first agent
to be specifically approved in the EU, USA, Canada and Japan for the treatment
of neuromyelitis optica spectrum disorder (NMOSD) in adults who
are aquaporin-4 water channel autoantibody (AQP4-IgG) seropositive and (in the
EU only) for those with a relapsing course of disease. In the phase III PREVENT trial, eculizumab significantly
reduced the risk of adjudicated relapse relative to placebo in patients with AQP4-IgG-seropositive NMOSD, approximately a quarter of whom did not
receive concomitant immunosuppressive therapies. The beneficial effect of
eculizumab was seen across all patient subgroups
analysed and was accompanied by improvements in
neurological and functional disability assessments, as well as generic health-related
quality of life measures; it was sustained through 4 years of treatment,
according to combined data from the PREVENT trial and an interim analysis of its
ongoing open-label extension study. The safety
profile of eculizumab in AQP4-IgG-seropositive NMOSD was consistent with that
seen for the drug in other approved indications. Thus, eculizumab provides an effective, generally well tolerated and approved
treatment option for this rare, disabling and potentially life-threatening
condition.
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