NewsHealthNew therapy for adults with paroxysmal nocturnal hemoglobinuria

New therapy for adults with paroxysmal nocturnal hemoglobinuria

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Alexion Pharma Italy, part of AstraZeneca, announces that the Italian Medicines Agency (Aifa) has approved the reimbursement of ravulizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (Epn) with hemolysis with clinical symptoms indicative of high disease activity , and also for clinically stable adult patients after being treated with eculizumab for at least the last six months. Ravulizumab is the first and only long-acting C5 complement inhibitor, offering comparable safety and efficacy to eculizumab, with infusions every 8 weeks versus every 2 weeks. The announcement was at the center of an online press conference promoted by Alexion Pharma Italy, to take stock of the new treatments and future standards of care that will allow a better quality of life for adult patients suffering from this rare haematological disease. “Paroxysmal nocturnal hemoglobinuria is an ultra-rare, debilitating and potentially fatal blood disease – says Simona Sica, director of Uoc Hematology and stem cell transplantation of the Agostino Gemelli Irccs Ucsc University Hospital in Rome -. We are facing a pathology. highly variable which involves delays in diagnosis and symptoms that can change over time. The quality of life of patients depends precisely on the symptoms caused by the disease and the speed with which it is diagnosed in order to be managed appropriately “. Epn, which affects 2-5 people per million inhabitants, is a disease characterized by complement-mediated destruction of red blood cells (haemolysis) and the risk of blood clots (thrombosis), which can occur throughout the body and cause organ damage and premature death. It can affect men and women of all ethnicities, origins and ages, without warning, with an average age of onset around 30. The pathology – it emerged from the webinar – is often not recognized, with delays in diagnosis ranging from one to more than five years. Prognosis can be poor in many cases, so timely and accurate diagnosis – as well as appropriate treatment – is critical to improving patient outcomes. “Anything that can improve the quality of life of people suffering from paroxysmal nocturnal hemoglobinuria makes us happy”, says Sergio Ferini Strambi, president of the Italian association of paroxysmal nocturnal hemoglobinuria. Ravulizumab works by blocking the C5 protein, an element of the terminal complement cascade, a part of the body’s immune system that is activated in an uncontrolled way in EPN. The action of ravulizumab is maintained for the eight weeks between administrations and eliminates the reactivation of haemolysis associated with an incomplete inhibition of protein C5.14,15 “Today, patients can count on a drug that reduces haemolysis in an extremely significant way – remembers Wilma Barcellini, head of Uos Pathophysiology of anemia, Fondazione Irccs Ca ‘Granda major hospital Policlinico di Milano -. However, the therapy involves an infusion every two weeks, which can be an important commitment for patients and caregivers . Having a drug such as ravulizumab available, which has a lower treatment load but the same efficacy and safety, is an important step forward for patients with Epn. ”The AIFA approval is based on the evaluation of a clinical presentation and financial report by Alexion, as well as on the evidence provided by clinical experts. Following the opinion of the Aifa Technical-Scientific Commission, the a determination of reclassification for the purposes of reimbursement of the drug for human use ravulizumab was published in the Official Gazette of January 5, 2022. “We are proud to have reached this important milestone – the comment of Anna Chiara Rossi, Vp & General Manager Italy, Alexion, AstraZeneca Rare Disease -. The reimbursement of ravulizumab represents for us the realization of what we work for every day: transforming the lives of people with rare diseases and disabling disorders, such as paroxysmal nocturnal hemoglobinuria. We are convinced that ravulizumab has the potential to become the new standard of care for the treatment of this pathology, because the benefit that this revolutionary treatment can bring in terms of quality of life for patients and those who care for it is great “. Ravulizumab is approved in the European Union for the treatment of adult patients with NPP with haemolysis and one or more clinical symptoms indicative of high disease activity, as well as for clinically stable adult patients after treatment with eculizumab for at least the previous six months. In Phase 3 clinical trials, in complement inhibitor-naïve patients with NPP and patients with stable NPP receiving eculizumab, treatment with intravenous ravulizumab every eight weeks has been shown to be non-inferior to intravenous treatment with eculizumab every 2 weeks. for all 11 endpoints. Ravulizumab has been awarded orphan drug designation for the treatment of patients with Epn in the United States and Japan. Ravulizumab acts on the immune system, which is why it can reduce its ability to fight infections. Due to its mechanism of action, the use of ravulizumab increases the patient’s susceptibility to meningococcal infection / sepsis (Neisseria meningitidis). Meningococcal infection due to any serogroup can occur. To reduce the risk of infection, all patients should be vaccinated against meningococcal infection at least two weeks before starting ravulizumab treatment, unless the risk of delaying ravulizumab therapy outweighs the risk of developing a meningococcal infection. . Patients starting ravulizumab treatment before 2 weeks of meningococcal vaccine administration should receive appropriate antibiotic prophylaxis up to 2 weeks after vaccination. In pregnant women – it was emphasized at the press conference – the use of the drug can be considered after an evaluation of the risks and benefits. It is also unknown whether ravulizumab is excreted in human milk. Preclinical reproductive toxicology studies, conducted in mice with the murine surrogate molecule BB5.1, did not show any adverse effects on the offspring deriving from the consumption of milk of the treated mothers. Since many medicinal products and immunoglobulins are excreted in human breast milk and due to the potential for serious adverse reactions in infants, breastfeeding should be discontinued during treatment with ravulizumab and for up to 8 months afterwards.

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