ASH: Novartis trumpets hospitalization data for newly minted sickle cell drug Adakveo

ORLANDO, FLORIDA—With an inaugural approval for targeted sickle cell disease therapy Adakveo under its belt, Novartis showed off a new analysis demonstrating the drug could cut down on patient hospitalizations.

Drawing on secondary endpoint data generated in the study that helped win Adakveo its FDA go-ahead last month, Novartis dove into its results to show that the drug—which helps prevent the organ-injuring vaso-occlusive crises that come along with the disease—could reduce the number of hospitalizations by 40%.

Adakveo also improved the proportion of patients who weren't hospitalized at all, Novartis showed Sunday at the American Society of Hematology annual meeting. About half of patients taking the drug never went to the hospital, versus about one-third of those on placebo, according to Andrew Cavey, who leads Novartis’ benign hematology development programs.

“We wanted to dig further” into the “numerically interesting results” turned up in the company’s phase 2 Sustain trial, Cavey said, and “all the data cuts point in the same direction.”

“The next phase of work is we need to generate more data,” he added.

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U.S. regulators green-lighted Adakveo in mid-November on the back of results showing the drug could slash the median annual rate of VOCs by 45% compared with placebo. And the Swiss drugmaker is counting the drug among a crop of recent approvals it thinks could pass the blockbuster benchmark.

“We worked extremely hard to get the approval fast,” Cavey said of the go-ahead, which came a couple of months before the FDA’s decision deadline. And the pre-ASH timing was a “lucky” result.

“There’s only, maybe with EHA, twice a year where the entire global community of hematologists is in one place,” he said. “The ability to disseminate the data, share the news—because this is a U.S. approval, to do so at ASH is that much more powerful.”