Alexion announced that Soliris (eculizumab), a first-in-class terminal complement inhibitor, has been granted an Orphan Drug designation by the FDA for the treatment of neuromyelitis optica (NMO), a life-threatening, ultra-rare neurological disorder. In patients with NMO, uncontrolled complement activation causes destruction of myelin-producing cells, leading to severe damage to the central nervous system, including the spinal cord and optic nerve.

The Phase 2 study was a single-arm, open-label, investigator-initiated trial in 14 women with severe, relapsing NMO who were treated for one year. The study met its primary efficacy endpoint, reduction in annualized relapse rate, with high degrees of clinical and statistical significance: a decline in the median annualized attack rate from 3 attacks per year pre-Soliris treatment to 0 attacks per year during 12 months of chronic Soliris treatment (P<0.0001). After 12 months of treatment, 86% (12 of 14) of these severely affected patients were completely attack-free.

Soliris is currently FDA-approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

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