UK-based pharmaceutical giant AstraZeneca Plc (AZ, NASDAQ: AZN) has announced that its rare disease drug Soliris (eculizumab) has received marketing approval in China for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in AQP4 antibody positive (Ab+) adults. This approval marks a significant expansion of Soliris’s application in the Chinese market, offering hope to patients with this rare and severe condition.
Soliris: A First-in-Class C5 Complement Inhibitor
Soliris, recognized as the global first-in-class C5 complement inhibitor, works by inhibiting the C5 protein in the terminal complement cascade. The drug’s approval for NMOSD treatment in China follows previous approvals for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (gMG), making it the first complement inhibitor available for NMOSD treatment in the country.
Clinical Trial Results and Treatment Benefits
The approval is based on the results of the Phase III PREVENT study, which demonstrated that eculizumab can effectively reduce the risk of recurrence in NMOSD. At week 48, 98% of patients in the eculizumab treatment group were recurrence-free, compared to 63% in the placebo group. The study showed a 94.2% reduction in the relative risk of recurrence with eculizumab (HR=0.058; 95% CI: 0.017-0.197; p<0.0001), with treatment benefits continuing until week 144. The 144-week follow-up data indicated that the recurrence-free rates were 96% in the eculizumab treatment group and 45% in the placebo group, respectively.
Future Impact on NMOSD Treatment
This approval not only expands the therapeutic options for NMOSD patients in China but also reinforces AstraZeneca’s commitment to addressing unmet medical needs in rare diseases. Soliris’s proven efficacy in reducing the risk of NMOSD recurrence is expected to significantly improve the quality of life for affected patients.-Fineline Info & Tech
