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Novartis' Adakveo becomes first FDA-approved P-selectin inhibitor for sickle cell disease

Published on 18/11/19 at 10:57am

The FDA has chosen to award Novartis’ Adakveo (crizanlizumab) with marketing authorisation, it has emerged, as a treatment to reduce the frequency of vaso-occlusive crises (VOCs) that occur as a result of sickle cell disease.

The approval covers patients aged at least 16 years old and marks the availability of the first FDA-authorised P-selectin inhibitor to treat the condition.

52-week trial data supporting the decision demonstrated that Adakveo “significantly lowered” the median annual VOC rate to 1.63 compared to 2.98 with placebo – a reduction of 45% – regardless of disease genotype and/or hydroxyurea use.

Furthermore, Novartis’ therapy dropped the median annual rate of days hospitalised to 4 compared to 6.87 with placebo, while it was found that 36% of Adakveo patients did not experience a VOC versus 17% with placebo. Additionally, median time to first VOC was 41. Months for Adakveo and 1.4 months for placebo.

"Patients with sickle cell disease often face unique challenges, and have long suffered silently through unimaginable pain crises,” said Beverley Francis-Gibson, President and CEO of the Sickle Cell Disease Association of America. "We are excited to have a new medicine that may help many of the thousands of people living with sickle cell disease by reducing the frequency of these potentially dangerous and painful episodes."

Dr Susanne Schaffert, President of Novartis Oncology, also commented: “The approval of Adakveo marks a new era in the treatment of sickle cell disease, a genetic condition that places an extraordinary burden of unpredictable pain crises on patients and their families. The stories we have heard from patients about their sickle cell pain crises are devastating. We are pleased to help reimagine medicine together with the sickle cell community and offer new hope for fewer VOCs.”

Matt Fellows

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