October 30, 2020: “Novartis announced that the European Commission (EC) has approved Adakveo (crizanlizumab) for the prevention of recurrent vaso-occlusive crises (VOCs), or pain crises, in patients with sickle cell disease aged 16 years and older.
Adakveo can be given as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.
Adakveo binds to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.
Sickle cell disease is considered a rare blood condition, affecting about 50,000 people in Europe.
“Data shows that nine out of ten people living with sickle cell disease experience one or more VOCs in a year, with a third of those crises leading to hospitalization, underscoring the significant unmet need among a vulnerable group of patients,” said Kees Roks, Head Region Europe, Novartis Oncology.
“Just one VOC could be catastrophic for the patient, so preventing these sudden, unpredictable and life-threatening events is hugely important. Today’s decision gives people living with sickle cell disease a chance to achieve that goal.”
The approval follows a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) in July based on results of the SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared with placebo (P=.010), equivalent to a 45% reduction. There was also a greater than two-fold increase in the proportion of patients with no VOCs who completed the study, compared to placebo.
Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea/hydroxycarbamide (HU/HC) use.
In the same study, Adakveo was shown to reduce the median annual rate of days hospitalized by 42% (4.0 days for Adakveo vs. 6.87 days for placebo).”
