Alexion gets approval for Soliris in Australia

US firm Alexion Pharmaceuticals says that the Australian government's  Therapeutic Goods Administration has approved the use of Soliris  (eculizumab) for the treatment of all patients in Australia with  paroxysmal nocturnal hemoglobinuria, a rare, debilitating and  life-threatening blood disorder defined by chronic red blood cell  destruction, or hemolysis. Soliris is the first therapy approved in  Australia for the treatment of PNH.

"Prior to this positive action by the government, there has not been an  approved, safe and effective therapy for the treatment of patients with  PNH in Australia," said Jeffrey Szer, director of clinical hematology at  the Royal Melbourne Hospital. "Soliris reduced hemolysis in every  patient treated in clinical studies. Regulatory approval is an important  step towards providing Australian patients with this debilitating and  life-threatening disease access to Soliris, the only approved  treatment. With access to Soliris, physicians will be able to prevent  the hemolysis that underlies the severe, progressive and  life-threatening consequences of PNH," he added.

The marketing application submitted to the Australian regulatory  authorities included safety and efficacy data from three multi-national  clinical studies: TRIUMPH, a placebo-controlled 26-week Phase III  evaluation involving 87 PNH patients, SHEPHERD, an open-label 52-week  Phase III trial involving 97 PNH patients and E05-001, a long-term  extension study. Soliris was approved by the US Food and Drug  Administration and the European Commission in 2007, and by Health Canada  in 2009, using data from the same studies. The drug is currently being  used to treat patients with PNH in more than 18 countries.