people with the disease. “The duration and severity of these pain crises worsens with aging. Often patients die during one of these crises,” said Dr. Biree Andemariam, chief medical officer of the Sickle Cell Disease Association of America.
Ameet Mallik, the Novartis’ head of U.S. oncology and blood disorders stated that VOCs send U.S. patients to emergency departments about 200,000 times per year. About 85% are hospitalized for days to a week, running up big bills.
The study showed Adakveo improved the proportion of patients who weren’t hospitalized at all. About 50% of the patients taking the drug never went to the hospital compared to about 30% of participants who were on placebo. This information was presented by Novartis at the American Society of Hematology annual meeting this past Sunday.
“We wanted to dig further” into the “numerically interesting results” turned up in the company’s phase 2 Sustain Trial and“all the data cuts point in the same direction,” Andrew Cavey, who leads Novartis’ benign hematology development programs.“The next phase of work is we need to generate more data,” he added.
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.
“We worked extremely hard to get the approval fast,” Cavey said of the go-ahead, which came a couple of