Alexion Pharmaceuticals announced Tuesday that the European Commission has expanded the label for Soliris (eculizumab) to include treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4)-antibody-positive with a relapsing course of the disease. The decision follows US approval of the drug in June for the same indication.
The EU decision was backed by data from the Phase III PREVENT trial, as well as an ongoing long-term extension study. PREVENT results, which were recently published in the NEJM, showed that 97.9%, 96.4% and 96.4% of Soliris-treated patients were relapse-free at 48 weeks, 96 weeks and 144 weeks, respectively, compared with rates of 63.2%, 51.9% and 45.4% for placebo. Further, among patients treated with Soliris without receiving other immunosuppressive therapies, all were relapse free at 48 weeks, and remained so through to 144 weeks, while in the placebo group relapse-free rates fell from 60.6%, 40.4% and 20.2% over the course of the three time periods.
Alexion R&D chief John Orloff stated "in a disease marked by unpredictable relapses that each have the potential for irreversible consequences, such as blindness or the inability to walk, the primary treatment goal is prevention of such attacks." He added "nearly all patients treated with Soliris were relapse-free in the Phase III study, demonstrating the potential of Soliris to change the treatment paradigm for this devastating disease."
Soliris was approved by EU regulators in 2011 for the treatment of atypical haemolytic uraemic syndrome. The drug is also cleared in Europe for paroxysmal nocturnal haemoglobinuria, as well as for certain patients with refractory generalised myasthenia gravis.
Sales of Soliris reached $980.8 million in the second quarter, up from $898.2 million in the prior-year period.
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