WASHINGTON -- Crizanlizumab, a P-selectin inhibitor, won FDA approval for treating vaso-occlusive crises in patients 16 and older with sickle cell disease, the .
To be sold by Novartis under the brand name Adakveo, the drug is the first to be approved that was specifically designed for this indication. Vaso-occlusive crises are a common complication of sickle cell disease, which affects about 100,000 people in the U.S., mainly those with African ancestry.
The approval was a long time coming, despite the drug being granted priority review and breakthrough therapy status. Results from the placebo-controlled phase II trial on which the FDA based its approval were reported 3 years ago.
In it, patients randomized to crizanlizumab had a median of 1.63 healthcare visits for vaso-occlusive crisis annually, compared to a median of 2.98 visits per year in the placebo group.
Moreover, the FDA noted, 36% of patients receiving the drug had no vaso-occlusive crises during the study, and it lengthened the median time to first crisis to 4.1 months, versus 1.4 months with placebo.
Adverse events seen in the drug's clinical studies included back pain, nausea, arthralgia, and fever, the FDA said. Crizanlizumab may cause platelet clumping in blood samples and interfere with automated platelet counts; the FDA advised clinicians to use citrate blood tubes and run tests quickly to minimize risk of inaccurate results.