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Novartis drug prevents pain due to sickle cell disease, post hoc analysis shows


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Novartis drug prevents pain due to sickle cell disease, post hoc analysis shows

Novartis AG said data from a mid-stage study showed that its investigational drug crizanlizumab prevented pain in more sickle cell disease patients than placebo.

A 52-week post hoc analysis of results from a phase 2 study, called Sustain, counted patients who experienced vaso-occlusive crises, or VOCs — a common, painful complication of sickle cell disease, linked to increased morbidity and mortality.

Of the 67 patients who received crizanlizumab, 35.8% did not experience a VOC after 52 weeks of treatment. Meanwhile, only 16.9% of the 65 patients who received placebo did not experience a VOC.

Novartis said all of the 132 evaluated patients had at least two VOCs in the year prior to the study — 62.9% had two to four VOCs, while 37.1% had five to ten VOCs.

Additionally, no new safety concerns were reported, with adverse events due to the treatment being similar between crizanlizumab- and placebo-treated patients.

These results were published in the American Journal of Hematology, Switzerland-based Novartis said in a news release.

"The insights gained from this analysis and others from the Sustain study, strengthen our belief that crizanlizumab may become an important new therapeutic option for sickle cell patients who continue to need step changes in medical innovation," said Samit Hirawat, the head of Novartis Oncology Global Drug Development.

The pharmaceutical giant also noted the limited treatment options for VOCs in the news release.

Novartis had reported positive 12-month results from the post hoc analysis of the Sustain trial in 2017, in which crizanlizumab delayed the occurrence of a sickle cell pain crisis by more months than placebo.

Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder typically have distorted red blood cells shaped like a sickle, or crescent. Patients experience pain when sickled red blood cells, which are stiff and inflexible, get stuck in small blood vessels. A particularly complication of sickle cell disease is high blood pressure in the blood vessels that supply the lungs.