Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization of 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.
“Sickle cell disease is a lifelong and devastating condition that affects patients, families and communities,” said Professor Jo Howard, Professor in Haemoglobinopathies, Guy’s and St Thomas’ NHS Foundation Trust in London. “Today’s positive opinion by CHMP means that we are one step closer to potentially having an important new medicine to treat thousands of vulnerable patients.” If approved by the European Commission, Adakveo will be the first targeted medicine available in Europe for the prevention of VOCs in patients with sickle cell disease.
Adakveo binds to P-selectin – a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion. Though considered a rare condition, tens of thousands of people in Europe have sickle cell disease.
The CHMP opinion was based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010), equivalent to a 45% reduction. Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea use. Additional results from the SUSTAIN study include:• A decrease in the median annual rate of days hospitalized to 4 vs 6.87 days when compared with placebo (a 42% reduction)• Median time to first VOC, 4.1 months for Adakveo vs 1.4 months for placebo.