The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for crizanlizumab (SEG101; Novartis) for the prevention of vaso-occlusive crises in patients with sickle cell disease.

Crizanlizumab is an investigational humanized monoclonal antibody that blocks P-selectin mediated multicellular adhesion; P-selectin is a major driver of the vaso-occlusive process in sickle cell disease.

The BLA is supported by data from the phase 2, multicenter, placebo-controlled, SUSTAIN trial that evaluated the safety and efficacy of crizanlizumab 5mg/kg IV infusion in preventing or reducing the occurrence of pain crises in 198 patients with sickle cell disease with or without hydroxyurea. 

Results showed crizanlizumab reduced the median annual rate of vaso-occlusive crises leading to healthcare visits by 45.3% vs placebo (1.63 vs 2.98, respectively; P =.010). In addition, a greater percentage of patients treated with crizanlizumab did not experience vaso-occlusive crises compared with placebo (36% vs 17%; P =.010). 

Moreover, compared with placebo, crizanlizumab was associated with a 3-fold longer median time to first vaso-occlusive crisis (4.07 vs 1.38 months; P <.001). Regarding safety, the most common treatment-emergent adverse events were back pain, nausea, pyrexia, and arthralgia.

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“The FDA’s decision to give crizanlizumab priority review reflects the impact that this medicine could have for the many thousands of US sickle cell adult patients who experience painful vaso-occlusive crises,” said John Tsai, MD, Head of Global Drug Development and Chief Medical Officer, Novartis. “We are looking forward to the opportunity, if crizanlizumab is approved, to reimagine medicine in sickle cell disease for patients who live with this condition every day of their lives.”

Crizanlizumab was granted Breakthrough Therapy designation in December 2018 and is currently being investigated in 4 additional clinical trials: SOLACE-adults, SOLACE-kids, STAND, and SUCCESSOR.

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