The first targeted therapy for the prevention of recurrent vaso-occlusive crises has been approved in Europe
Novartis announced today that the European Commission (EC) has approved Adakveo® (crizanlizumab) for the prevention of recurrent vaso-occlusive crises (VOCs) or "painful crises" in patients with sickle cell disease from 16 years of age1. Crizanlizumab can be administered as add-on therapy to hydroxyurea / hydroxycarbamide (HU / HC) or as monotherapy in patients for whom HU / HC is inappropriate or inadequate.
Sickle cell anemia is considered a rare haematological disease, affecting approximately 50.000 people in Europe.4,5 Crizanlizumab binds to P-selectin, a cell adhesion protein that plays a central role in multicellular interactions that can lead to vaso-occlusion. 2,3
"The European approval of this therapeutic option represents very important news for patients with sickle cell anemia who after many years have at their disposal a new treatment tool capable of improving their quality of life - commented Lucia De Franceschi, Associate Professor of Internal Medicine at the University of Verona -. Only crizanlizumab, thanks to its peculiar mechanism of action, is in fact able to act on the chronic inflammatory vasculopathy which underlies the numerous clinical complications present both in adolescents and adults but also in children.
Furthermore, crizanlizumab has a unique profile that makes it very interesting for us clinicians because it could help us manage even those patients who have failed or do not accept gold standard therapies for sickle cell anemia such as hydroxyurea or chronic transfusion regimens. "
“We are proud that our long-term commitment to research and development of innovative solutions for hereditary blood diseases brings about an important change in the way we treat sickle cell anemia - says Luigi Boano, General Manager of Novartis Oncology Italy. This milestone will allow us to make available to patients the first targeted drug for the prevention of recurrent vaso-occlusive crises, which disrupt patients' lives from a physical, social and emotional point of view and can degenerate into acute and long-term complications ".
The approval follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP), issued in July based on the results of the SUSTAIN clinical study, which showed that crizanlizumab significantly reduced the median annual rate of VOC at 1,63, compared to 2,98 for placebo (P = .010), equal to a reduction of 45%. There was also a more than double increase in the percentage of patients without VOCs who completed the study, compared to placebo. Reductions in VOC frequency have been observed among patients regardless of sickle cell anemia genotype and / or use of hydroxyurea / hydroxycarbamide (HU / HC). In 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) 6.
About crizanlizumab
Crizanlizumab - formerly known as SEG101 - is indicated for the prevention of recurrent VOCs in patients with sickle cell disease from 16 years of age. It can be given as add-on therapy to HU / HC, or as monotherapy in patients for whom HU / HC is inappropriate or inadequate. It is the first and only targeted biologic drug that works by binding to P-selectin, a cell adhesion protein that plays a central role in multicellular interactions that can cause vaso-occlusion in sickle cell disease. By binding to P-selectin on the surface of the endothelium and activated platelets, crizanlizumab blocks interactions between endothelial cells, platelets, red blood cells and leukocytes.
Crizanlizumab is now approved in 36 countries around the world, including the United States and EU member states.
Sickle cell anemia
Sickle cell anemia is one of the most common genetic blood disorders in the world.7 It is a chronic, permanent and debilitating disease of varying clinical severity.8 Sickle cell anemia is characterized by the alteration of the shape and physical properties of red blood cells and by a greater adhesiveness of the different blood cells than usual.8 In certain situations, these cells are activated and adhere to each other and the internal wall of the blood vessels, forming agglomerates (clusters) 9,10 which in turn can slow down, blocking and reducing the flow of blood and oxygen, causing damage to blood vessels and organs.1,11 This consequently leads to recurrent and unpredictable acute crises of painful vascular occlusion, also called vaso-occlusive crises1 responsible for organ damage. The painful crises typical of sickle cell anemia disrupt the lives of patients physically, socially and emotionally: in addition, they involve acute, and long-term complications.12
People with sickle cell disease have inherited two abnormal copies of the hemoglobin gene from their parents.8 Those with "sickle cell trait" (also called "carriers") have inherited one abnormal gene and one normal gene.9 The sickle cell trait can be asymptomatic, but individuals with the disease can pass it on to their children.9 If both parents have the trait, their children are 25% likely to have sickle cell disease, 50% to have sickle cell trait, and 25% have two normal genes or have no sickle cell trait or sickle cell anemia.13 A simple blood test can determine if a person has sickle cell trait.13
Disclaimer
This press release contains some guidance that may not reflect future results. In the event that one or more of these risks or uncertainties materialize, or in the event that the assumptions that led to the advances were to be incorrect, the actual results could be different from those described here as anticipated, believed, estimated or expected. The information contained in this press release is a translation of the press release issued by Novartis AG on 26/06/2020.
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Novartis is interpreting medicine in a new way to improve and extend people's lives. As a global leader in the pharmaceutical industry, we use innovative scientific and digital technologies to create transformative therapies in areas with significant medical needs. Committed to discovering new drugs, we are consistently among the top companies in the world in investing in research and development. Novartis products reach more than 800 million people globally and we work to find innovative ways to expand access to our latest treatments. Worldwide, approximately 110.000 people of 145 different nationalities work for Novartis. Further information on www.novartis.it and www.novartis.com. @NovartisItalia is also on Twitter and LinkedIn.
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