Phase 3 Trial
Results from the Phase 3 PREVENT study for neuromyelitis optica spectrum disorder (NMOSD) were extremely positive. 98% of patients in the treatment group did not experience a relapse/attack of the condition. Considering the fact that each attack can further disability for a patient (including paralysis or blindness), minimizing chance of relapse is a critical component to treatment. The treatment examined in this trial is called Soliris (otherwise called eculizumab).
The Phase 3 trial was both randomized and double-blind. 96 patients were given Soliris and 47 patients were given placebo. At the 48 week mark, the vast majority of Soliris-treated patients had not experienced a relapse compared to just 63% of those who received placebo.
A quarter of the patients in the trial were not given immunosuppressive therapies. Of these individuals who only received Solaris, 100% did not relapse. Of those in this group who received placebo, 61% of patients did not relapse. The effects of Solaris for all patients were observed for 144 weeks.
Additionally, the safety profile of this drug stayed consistent with previous studies with only mild adverse events reported for most patients. Results from this trial were published in The New England Journal of Medicine.
As a result of this investigation, Japan’s Ministry of Health, Labour and Welfare has announced that they have approved Soliris for marketing for anti-aquaporin antibody positive NMOSD patients as a means of preventing relapse.
This is amazing news for patients in Japan! It will be exciting to see the future of this therapy, especially as it begins to become more widespread.
You can read more about this new approval here.