Soliris eculizumab regulatory update

The U.K.’s NICE issued final draft guidance recommending Orphan drug Soliris eculizumab from Alexion to treat atypical hemolytic uremic syndrome (aHUS). The guidance is in line with draft guidance issued in September and contingent on measures to reduce the drug’s impact on NHS’s budget. Those measures include the coordination of Soliris use through an expert center; monitoring systems to record the number of patients diagnosed with aHUS and those who have received Soliris as well as the dose and duration of treatment; a national protocol for beginning and ending treatment for clinical reasons; and a research program with “robust” methods to evaluate when beginning treatment and when dose adjustment might occur (see BioCentury, Sept. 8, 2014). ...