Last Updated on January 16, 2019 by Sultan Beardsley
NVS receives Breakthrough Therapy Designation (BTD) for crizanlizumab (SEG101) for the prevention of vaso-occlusive crises in sickle cell disease
- Crizanlizumab is under development as a monthly infusion treatment for preventing pain in Crises (aka vaso-occlusive crises, or VOCs) in individuals with sickle cell disease (SCD)
- Sickle cell Crises is associated with increased morbidity and mortality, and is stimulated by multi-cell adhesion blocking blood flow.
- The FDA filing for crizanlizumab is expected in the 1st half of 2019
- Crizanlizumab was granted BTD for patients of all genotypes with sickle cell disease for the prevention of pain associated with VOCs.
- VOCs (sickle cell pain crises) are extremely painful and unpredictable occurrences that can lead to serious chronic and acute complications.
- Sickle cell pain crises happens when blood cells stick to each other and blood vessels causing blockages. By prevent or reducing the prevalence of blood cells sticking together NVS can potentially reduce the frequency that patients experience VOCs.
- On average each sickle cell patient is burdened by $1 million in healthcare related costs. The estimated annual cost for adults is over $30,000
- NVS is up 1.30% premarket.
Statement by Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development:
“Painful sickle cell crises matter because they can disrupt patients’ lives, and often require hospital visits and medical attention…We look forward to working closely with the FDA over the coming months toward making crizanlizumab, a therapy that has the potential to prevent sickle cell pain crises, available in the US as soon as possible.”NVS press release
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