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Crizanlizumab: First Approval
1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.
Insights
Crizanlizumab is a new treatment approved to prevent vaso-occlusive crises in sickle cell disease patients. This monoclonal antibody targets P-selectin, offering a novel approach to managing this condition.
Area of Science:
- Hematology
- Immunology
- Pharmacology
Background:
- Sickle cell disease (SCD) is a genetic blood disorder characterized by vaso-occlusive crises (VOCs).
- P-selectin plays a crucial role in the pathophysiology of VOCs by mediating cellular adhesion.
- Existing treatments for SCD focus on managing symptoms and preventing complications.
Purpose of the Study:
- To summarize the development milestones of crizanlizumab.
- To highlight the regulatory approval of crizanlizumab for preventing VOCs in sickle cell disease.
Main Methods:
- Crizanlizumab is a monoclonal antibody that targets P-selectin.
- It blocks the interaction between P-selectin and its ligand, P-selectin glycoprotein ligand-1.
- Development involved preclinical studies and clinical trials to assess efficacy and safety.
Main Results:
- Crizanlizumab received FDA approval in November 2019 for reducing VOC frequency in SCD patients (≥16 years).
- The drug is under regulatory review in the EU for the same indication.
- Investigational use in myelofibrosis with ruxolitinib is also being explored.
Conclusions:
- Crizanlizumab represents a significant advancement in the management of sickle cell disease.
- Its approval marks a milestone in targeted therapy for preventing VOCs.
- Further research is ongoing for other indications and patient populations.
Abstract:
Crizanlizumab (Adakveo®; crizanlizumab-tmca) is an intravenously administered monoclonal antibody developed by Novartis Pharmaceuticals for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease. Crizanlizumab binds to P-selectin, thereby blocking its interaction with P-selectin glycoprotein ligand-1. In November 2019, crizanlizumab received its first global approval in the USA, where it is indicated to reduce the frequency of VOCs in adults and paediatric patients aged ≥ 16 years with sickle cell disease. The drug is also under regulatory review in the EU for the prevention of VOCs in patients with sickle cell disease. The use of crizanlizumab (in combination with ruxolitinib) in myelofibrosis is also being evaluated in Australia, Spain, Germany and Hungary. This article summarizes the milestones in the development of crizanlizumab leading to this first approval for the reduction of VOCs in patients with sickle cell disease.
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