Review shows approved treatment sharply cuts relapse risk in NMOSD
More than 95% of patients receiving Soliris remained relapse-free during follow-up
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Treatment with Soliris (eculizumab) substantially reduces the risk and frequency of relapses in adults with neuromyelitis optica spectrum disorder (NMOSD) who test positive for antibodies against the aquaporin-4 (AQP4) protein.
That’s according to a systematic review of published studies reporting results from clinical trials testing Soliris in this patient population. All of the studies included in the final analysis concerned the global Phase 3 PREVENT clinical trial (NCT01892345), whose top-line data supported Soliris’ approvals for AQP4-related NMOSD, and its open-label extension (OLE) study (NCT02003144).
The pooled findings support Soliris “as an effective and well-tolerated option for preventing relapses and managing long-term disease in patients with [AQP4-related] NMOSD,” researchers wrote. “Nevertheless, the lack of [additional appropriately-controlled trials], small sample sizes, [variability] in follow-up periods, and limited data on long-term safety and cost-effectiveness require additional studies.”
The study, “A systematic review and meta-analysis of the efficacy and safety of eculizumab in AQP4-IgG–positive neuromyelitis optica spectrum disorder,” was published in Multiple Sclerosis and Related Disorders.
Soliris designed to block key protein
NMOSD is an autoimmune disease marked by inflammation and damage in the nervous system, particularly the spinal cord and optic nerves, which carry visual signals from the eyes to the brain. In most patients, self-reactive antibodies against AQP4, a water-channel protein found mainly on support cells in the brain and spinal cord, drive the disease.
When anti-AQP4 antibodies bind to these cells, they activate the complement system, a part of the immune response, ultimately contributing to nerve tissue damage. Because each relapse can cause permanent neurological damage, preventing relapses is considered the primary goal of NMOSD treatment.
Soliris is an infusion therapy approved for adults with NMOSD who are positive for anti-AQP4 antibodies. It’s designed to block the complement protein C5, thereby suppressing complement activation and reducing the risk of NMOSD relapses.
“The objective of this [study] is to systematically review and meta-analyze the effectiveness and safety of [Soliris] therapy in NMOSD patients with [anti-AQP4] antibodies” within clinical trials, the researchers wrote.
Soliris reduced risk of relapse by 93% compared with a placebo
From the more than 1,000 studies initially screened, three met inclusion criteria and were included in the final meta-analysis.
The first study reported data from the main portion of PREVENT, which tested Soliris against a placebo in 143 adults with AQP4-related NMOSD, while the second study represented a subgroup analysis of data from that placebo-controlled portion. The third study described data from PREVENT and its OLE study, in which 119 participants received Soliris for up to 5.5 years.
According to the pooled analysis, Soliris significantly reduced the risk of relapse by 93% compared with the placebo. The treatment was also associated with a 85% lower annualized relapse rate, or the average number of relapses a patient experienced in one year. In addition, more than 95% of patients receiving Soliris remained relapse-free during follow-up.
[Soliris] is generally very beneficial from a clinical standpoint in the prevention of disease activity as well as neurological stability in [anti-AQP4 antibody] positive patients with NMOSD.
Patient disability, as assessed with the validated Expanded Disability Status Scale (EDSS), generally remained stable, with some investigations reporting “a slight improvement in EDSS scores [and] others [reporting] stable scores without further deterioration,” the researchers wrote.
“Although there was no significant change in the EDSS score, maintaining its stability is important in a condition where relapse is associated with irreversible disabilities,” they added.
Regarding safety, data showed no significant differences in adverse events between Soliris and control groups. Commonly reported adverse events included headache, upper respiratory infection, common cold, and infusion-related reactions, most of which were mild or moderate in severity.
“[Soliris] is generally very beneficial from a clinical standpoint in the prevention of disease activity as well as neurological stability in [anti-AQP4 antibody] positive patients with NMOSD,” the researchers concluded.
They emphasized, however, that there’s “still a need for additional [appropriately controlled] clinical trials … with extended observation periods to assess any potential safety issues as well as cost-effectiveness and compare efficacy in relation to other … medications.”
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