Regulatory, Results

Novartis builds its case for a new sickle cell drug, eyeing a delayed FDA filing in ‘19

Researchers at Novartis have taken another crack at the data they collected in a Phase II study of their sickle cell disease drug crizanlizumab, building their case ahead of a looming regulatory filing.

Checking on the rate of disease-related pain crises among patients, Novartis scored their drug with 35.8% of the patients on drug avoiding a crisis during the previous year. That’s more than twice the 16.9% rate tracked in the placebo arm.

All of the patients in the SUSTAIN study had experienced at least 2 episodes of what’s called a vaso-occlusive crisis in the year before they joined the study, putting them at high risk for more.

Abdullah Kutlar

“The unpredictable, intense painful crises that patients with sickle cell disease experience are the hallmark of the disease and the primary cause of hospitalizations in this patient population,” said Abdullah Kutlar, the primary author of the SUSTAIN analysis. “I am encouraged that results from this post hoc analysis of SUSTAIN study data found that crizanlizumab could substantially delay or prevent these crises, which also may mean less organ damage in the long run.”

These pain crises occur when sickle-shaped cells block the flow of blood in small blood vessels. And it’s the most obvious symptom of the disease. Novartis picked up the drug in 2016 in their acquisition of Selexys Pharmaceuticals in a deal valued at up to $665 million.

Outlining the response in the high-dose arm 2 years ago, investigators reported in the New England Journal of Medicine the median rate of crises per year was “1.63 with high-dose crizanlizumab versus 2.98 with placebo (indicating a 45.3% lower rate with high-dose crizanlizumab, P=0.01). The median time to the first crisis was significantly longer with high-dose crizanlizumab than with placebo (4.07 vs. 1.38 months, P=0.001), as was the median time to the second crisis (10.32 vs. 5.09 months, P=0.02). The median rate of uncomplicated crises per year was 1.08 with high-dose crizanlizumab, as compared with 2.91 with placebo (indicating a 62.9% lower rate with high-dose crizanlizumab, P=0.02).”

The low dose did not deliver a statistically significant response.

Novartis says now that it’s looking at a 2019 FDA filing, which represents a delay from the 2018 timeline execs had provided earlier after the initial readout arrived.


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