Novartis drug cuts serious complications in sickle cell disease

Novartis’ developmental sickle cell disease drug crizanlizumab cut occurrences of a serious complication of the disease that can lead to irreversible or fatal organ damage, according to mid-stage data.

Vaso-occlusive crisis (VOC) is a common and painful complication of the disease, where circulation of blood vessels is obstructed by sickled red blood cells.

The restriction in the oxygen supply initially causes pain but in severe cases, organs are damaged. This can potentially cause stroke, the death of lung tissue, and death in extreme cases.

Results from an analysis of the phase II SUSTAIN study showed that more patients treated with crizanlizumab did not experience VOC compared with placebo (35.8% vs. 16.9%), among patients with a history of 2-10 VOCs in the previous year.

The post hoc analysis reviewed 52-week results from 132 patients, including 67 treated with 5 mg/kg crizanlizumab and 65 who received placebo.

All evaluated patients had a history of at least 2 VOCs in the year prior to the study, with 62.9% having experienced 2-4 events and 37.1% with 5-10 events.

The most common genotype in the disease, homozygous haemoglobin S (HbSS), was identified in more than 70% of SUSTAIN patients and patients with this genotype were evenly distributed between study arms.

Of the subgroups evaluated, a considerable number of patients across multiple subgroups treated with crizanlizumab did not experience a VOC compared with those treated with placebo.

No new safety concerns emerged in the post hoc analysis as adverse events attributed to treatment were similar between the crizanlizumab and placebo arms across all subgroups.

Novartis acquired crizanlizumab when it bought Oklahoma-based biotech Selexys in a deal in 2016 worth up to $665 million, if all development and sales goals are met. The Swiss pharma obtained the right to buy Selexys and the drug formerly known as SelG1 in 2012.

Novartis expects to file the drug with the FDA in 2019 and discussions with other health authorities are ongoing.

Samit Hirawat, head of Novartis Oncology global drug development, said: “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.”