Rare diseases, new drug for paroxysmal nocturnal hemoglobinuria is available in Italy

The orphan drug pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (Epn) has received reimbursement from the Italian Medicines Agency for adult patients with this ultra-rare chronic hematological disease, who remain anemic after treatment with a C5 inhibitor for at least 3 months. This was announced by the biopharmaceutical company Sobi. Pegcetacoplan (Aspaveli *) has received orphan drug designation, granted to therapies that treat a serious disease affecting fewer than five in 10,000 people in the European Union and providing a significant benefit over existing treatments. 5 and 2 people per million inhabitants: it is a rare, chronic and life-threatening blood disease, in which the uncontrolled activation of complement (a component of our immune system) leads to the destruction of red blood cells carrying oxygen through the ‘intravascular and extravascular hemolysis. Characterized by persistently low hemoglobin, EPN may require frequent transfusions and lead to debilitating symptoms such as severe fatigue caused by anemia, difficulty concentrating, abdominal pain, dyspnoea. The most serious complication is thrombosis, which is the formation of clots inside the blood vessels, especially the veins. “The main goal of every hematologist is to offer their patients an effective and safe treatment option that can guarantee them a good quality of life. For this reason, from today we are happy to have pegcetacoplan available, which by inhibiting the complement at the C3 level, offers a response to those patients who remain anemic despite treatment with C5 inhibitors “, explains Wilma Barcellini, head of the Uos Physiopathology of anemias at the Irccs Ca ‘Granda Foundation, Maggiore Policlinico hospital in Milan. The approval of pegcetacoplan is based on the results of the phase 3 Pegasus study (published in the New England Journal of Medicine), which assessed the efficacy and safety of the drug with respect to to eculizumab at 16 weeks in adults with PNH who had persistent anemia despite treatment with eculizumab. A mean increase of 3.84 g / dL in hemoglobin was observed with pegcetacoplan compared to the group of patients treated with eculizumab. and safety was further confirmed by positive results at 48 weeks recently published in Lancet Haemathology. ” Pegcetacoplan represents a new class of complement drugs after more than a decade – says Giampiero Marra, Medical Director Sobi Italy, Greece, Cyprus and Malta – The Italian reimbursement less than 8 months from the European approval demonstrates the general expectation by the scientific community of a therapeutic option that can respond to needs that are still unsatisfied despite the treatments available “.