Sickle cell anemia, in Italy the first therapy that prevents vaso-occlusive crises

Novartis announced today that the Italian Medicines Agency has approved reimbursement Adakveo * (crizanlizumab) for the prevention of recurrent vaso-occlusive (VOC) crises in patients with sickle cell disease, aged 16 years and older, who have submitted at least two Vocs in the previous 12 months. Crizanlizumab – to which Aifa has recognized the requirement of conditioned innovation – can be administered as an add-on therapy to hydroxyurea / hydroxycarbamide (HU / HC) or as monotherapy in patients for whom HU / HC is inappropriate or inadequate, where for inappropriate / inadequate we mean insufficient efficacy or the presence of tolerability problems, insufficient compliance. Sickle cell anemia is one of the most common genetic blood diseases in the world, as well as being a chronic, permanent and debilitating disease of varying clinical severity. In Europe it affects about 50 thousand people but in Italy it is considered a rare haematological pathology due to the difficulty of tracing and diagnosis: the cases recorded with a serious clinical picture are about 2,500-2,800 although, according to experts, there is an important undeclared twice as many patients. Crizanlizumab, designated orphan drug, binds to P-selectin, a cell adhesion protein that plays a central role in multicellular interactions that can result in vaso-occlusion. Vaso-occlusive crises (Vocs) are serious, unpredictable events and can represent real health emergencies due to their rapid evolution and high mortality. As evidenced by the international research ‘Sway’ (Sickle Cell World Assessment Survey), 91% of patients report at least one vaso-occlusive crisis in the 12 months prior to the survey. Overall, patients reported an average of 5.3 Vocs in the previous 12 months, most of which managed with medical intervention. “The approval of the reimbursement in Italy of the first targeted therapy for recurrent vaso-occlusive crises represents very important news both for the patient community and for clinicians – commented Lucia De Franceschi, associate professor of internal medicine, Aoui Verona and University of Verona – Crizanlizumab, thanks to its peculiar mechanism, acts directly on chronic inflammatory vascular disease, which is the basis of the numerous clinical complications of patients with sickle cell syndrome. Furthermore, crizanlizumab has a unique profile that makes it very interesting for we doctors because it could help us manage even those patients who have failed or do not accept therapies considered standard. “From a narrative medicine project of the Istud Foundation, promoted by Novartis, it also emerged clearly that sickle cell anemia has a significant impact on the quality of life of patients, from the affective to the pro sphere fessional or scholastic: in the periods in which symptoms occur, in fact, people with sickle cell anemia find it difficult to concentrate and carry out their study or work activities. On average, they are absent 39 days from their workplace or school. Other activities of daily life, such as shopping for example, are also often affected by the disease. In addition, more than 50% of people with sickle cell anemia carry out transfusions at least once a month. However, despite these difficulties, the narratives also show the great desire of these people to cope with the care and the right assistance. Compared to the diagnosis, in 43% of cases it is carried out and communicated by a center other than the one in which one is currently being treated, often represented by a pediatric center. However, there are those who arrived at the diagnosis after a pilgrimage between several treatment centers (28%). In a few cases, the diagnosis was made following a traumatic event during childbirth, or in the same center where one is still being treated. Furthermore, in one third of the reported cases (33%) it is specified how initially a diagnosis other than sickle cell anemia was made, confused with other forms of anemia – in particular Beta Thalassemia – or other conditions such as pain in growth and rheumatism . “We are really proud to have been able to make available to patients and the medical profession the first targeted therapy for recurrent vaso-occlusive crises in sickle cell anemia – says Luigi Boano, General Manager of Novartis Oncology Italy – This result testifies how our continuous commitment to the research and development of innovative solutions is able to bring about an important change in the management of patients with this pathology, which is currently an orphan of therapeutic solutions “. The approval of reimbursement by Aifa follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency and the same approval by Ema, issued in October 2020 based on the results of the clinical study ‘Sustain’, which demonstrated that crizanlizumab significantly reduced the median annual rate of Voc by 45% (1.63, compared with 2.98 for placebo (P = .010). Voc among patients regardless of sickle cell disease genotype and / or use of hydroxyurea / hydroxycarbamide. There was a more than double increase in the percentage of patients without Voc who completed the study compared to placebo. of the same study, crizanlizumab was shown to reduce the median annual rate of hospitalization days by 42% (4.0 days for crizanlizumab vs 6.87 days for placebo). Based on clinical trials, crizalizumab it also has a favorable safety profile: in fact, similar adverse events were highlighted between the patients treated with the therapy and the placebo group. Crizanlizumab is now approved in 36 countries around the world, including the United States and EU member states.