EMA extends market authorisation for Alexion Pharma’s eculizumab
London – The European Medicines Agency has extended the therapeutic range of Soliris (eculizumab), a humanised monoclonal anti-C5 antibody that blocks terminal activation of the complement cascade of the human immune system. According to the Agency’s CHMP, treatment of paediatric and adult patients with the rare disease atypical haemolytic uraemic syndrome (aHUS) will be included provided that special precautionary measures are taken to prevent meningococcal infections. aHUS is characterised by hemolysis and thrombocytopenia, resulting in the formation of multiple blood clots in small blood vessels (so-called thrombotic microangiopathy, TMA). Patients with TMA require dialysisto prevent permanent kidney damage. The approval of eculizumab is based on 2 prospective open-label studies in adults and children. After treatment with Soliris 16 out of 20 adult patients achieved a stable platelet count over 12 weeks and no longer required plasma exchange or dialysis. Additionally renal function significantly improved. In children, the improvement was even better, with 93% of patient platelet count normalised. Most frequent adverse effects were hypertension, upper respiratory tract infection, and diarrhoea. In Europe, patients must be vaccinated with a tetravalent jab against meningococcus, because Soliris increases patient’s susceptibility to infections with Neisseria meningitidis.